Prof Adrian Thrasher

Photo

Personal Profile

Name: Adrian Thrasher Email: a.thrasher@ucl.ac.uk
Title: Prof Tel: 0044 207 905 2660
Department: ICH - Molecular Immunology Unit Fax: 0044 207 905 2810
Position: Professor in Paediatric Immunology and Wellcome Trust SRF Address: Molecular Immunology Unit, UCL Institute of Child Health, 30 Guilford Street, London, WC1N 1EH
Research Domain: Infection, Immunology & Inflammation, Neuroscience, Personalised Medicine, Populations & Lifelong Health, Reproduction & Development Web Page:  

Profile

Research Description

Conducting trials of somatic gene therapy for various forms of PID including SCID-X1, CGD, ADA-SCID, and WAS. Other research interests include the pathophysiology of primary immunodeficiency syndromes especially WAS, the actin cytoskeleton in haematopoietic cells (with Dr Siobhan Burns), the development of somatic gene therapy (with Professor Bobby Gaspar, Professor Christine Kinnon, Dr Waseem Qasim, and for ocular disease with Professor Robin Ali), and thymus transplantation (with Dr Graham Davies).

Research Activities

Axonal regeneration

Development of novel therapies for the treatment of retinal degeneration

Gene and Cellular Therapy of Haematological Malignancies

Gene therapy for haematological disorders

Immunopathogenesis of paediatric autoimmune diseas

Paediatric Neurology

Primary Immunodeficiency

T cell therapies

gene therapy

Education Description

UCL Collaborators

Prof Steve Bolsover; Prof Hans Stauss; Prof William Richardson; Prof David Becker; Prof Paul Griffiths; Dr Mike Hubank; Prof Jane Sowden; Dr Waseem Qasim; Prof Robin Ali; Prof Michael Duchen; Prof Patrick Anderson; Prof Mary Reilly; Prof William Mckenna; Prof Edward Tuddenham; Dr Paul Brogan; Prof Amit Nathwani; Prof Bobby Gaspar; Dr Jeremy Cook; Dr Karl Peggs; Prof Samuel Janes; Dr Mona Bajaj-Elliot; Prof Lucy Wedderburn; Dr Emma Morris; Prof Francesco Muntoni; Prof Perry Elliott; Prof Jenny Morgan

External Collaborators

Publications

    2014

    • Burns SO, Plagnol V, Gutierrez BM, Al Zahrani D, Curtis J, Gaspar M, Hassan A, Jones AM, Malone M, Rampling D, McLatchie A, Doffinger R, Gilmour KC, Henriquez F, Thrasher AJ, Gaspar HB, Nejentsev S (2014). Immunodeficiency and disseminated mycobacterial infection associated with homozygous nonsense mutation of IKKβ(⋆). J Allergy Clin Immunol, , - . doi:10.1016/j.jaci.2013.12.1093
    • Chandrashekran A, Sarkar R, Thrasher A, Fraser SE, Dibb N, Casimir C, Winston R, Readhead C (2014). Efficient generation of transgenic mice by lentivirus-mediated modification of spermatozoa. FASEB JOURNAL, 28(2), 569 - 576. doi:10.1096/fj.13-233999
    • Pike-Overzet K, Fibbe WE, Zwaginga J-J, Staal FJT, Bredius RGM, Lankester A, Hoeben RC, Meij P, Baum C, Schambach A, Cavazzana M, Lagresle-Peyrou C, Driessen G-J, Gaspar HB, Thrasher A, Van Dongen JJM (2014). Successful RAG1-SCID gene therapy depends on the level of RAG1 expression. Journal of Allergy and Clinical Immunology, 134(1), 242 - 243. doi:10.1016/j.jaci.2014.04.033
    • Burns SO, McLatchie A, Gutierrez BM, Hassan A, Jones AM, Malone M, Rampling D, Gilmour KC, Henriquez F, Thrasher AJ, Gaspar HB, Plagnol V, Al Zahrani D, Curtis J, Gaspar M, Doffinger R, Nejentsev S (2014). Immunodeficiency and disseminated mycobacterial infection associated with homozygous nonsense mutation of IKKβ. Journal of Allergy and Clinical Immunology, 134(1), - . doi:10.1016/j.jaci.2013.12.1093
    • Carbonaro DA, Jin X, Geiger S, Cooper A, Kaufman ML, Hollis RP, Senadheera S, Fu P-Y, Sahaghian A, Chan RY, Kohn DB, Zhang L, Montiel-Equihua C, Carmo M, Blundell MP, Thrasher AJ, Gaspar HB, Fairbanks L, Sebire NJ, Wang X, Cornetta K (2014). Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency. Molecular Therapy, 22(3), 607 - 622. doi:10.1038/mt.2013.265
    • Pettrova A, Di W-L, Harper J, Chan L, Farzaneh F, Thrasher A, Qasim W (2014). GMP-compliant SPINK5 lentiviral vector corrects keratinocyte stem cells isolated from patients with Netherton syndrome.
    • Georgiadis C, Almarza D, Thrasher A, Di W-L, Qasim W (2014). Zinc finger nuclease-mediated modelling of COL7A1 deficient RDEB.
    • Gaspar B, Buckland K, Rivat C, Himoudi N, Gilmour K, Booth C, Xu-Bayford J, Cornetta K, Kohn D, Carbonaro D, Paruzynski A, Schmidt M, Thrasher A (2014). Immunological and metabolic correction after lentiviral vector mediated haematopoietic stem cell gene therapy for ADA deficiency.
    • Carmo M, Calero-Garcia M, Thrasher AJ, Gaspar HB (2014). Development of gene therapy for hemophagocytic lymphohistiocytosis (HLH) due to perforin deficiency - corrected T cell transplant as control therapy.
    • Abdul-Razak H, Rocca C, Howe S, Alonso-Ferrero ME, Gabriel R, Bartholomae C, Gan CHV, Garin M, Molina Estevez FJ, Roberts A, Blundell M, Wang J, Prakash V, Gueenechea G, Holmes M, Gregory P, Kinnon C, von Kalle C, Schmidt M, Antonio Bueren J, Thrasher A, Yanez-Munoz R (2014). Gene editing of mutated Prkdc gene in classical scid mouse.
    • Bouma G, Carter NA, Recher M, Malinova D, Adriani M, Notarangelo LD, Burns SO, Mauri C, Thrasher AJ (2014). Exacerbated experimental arthritis in Wiskott-Aldrich syndrome protein deficiency: Modulatory role of regulatory B cells.. Eur J Immunol, , - . doi:10.1002/eji.201344245
    • Georgiadis C, Almarza D, Abdul-Wahab A, McGrath J, Harper J, Thrasher A, Qasim W, Di W-L (2014). The psychophysiological stress response in psoriasis and rheumatoid arthritis. doi:10.1111/bjd.12697
    • Petrova A, Di W-L, Qasim W, Chan L, Farzaneh F, Thrasher A, Harper J (2014). Characterization of SPINK5-transduced keratinocytes under good manufacturing practice conditions for Netherton syndrome.
    • Shangaris P, Loukogeorgakis SP, Blundell MP, Petra E, Ramachandra D, Maghsoudlou P, Urbani L, Bertin E, Thrasher A, De Coppi P, David AL (2014). Congenic Amniotic Fluid Stem Cells Show Stable Long-Term Engraftment in the Haematopoietic System after In Utero Transplantation. REPRODUCTIVE SCIENCES, 21(3), 83A - 83A.
    • Mukherjee S, Thrasher AJ (2014). Gene correction of induced pluripotent stem cells derived from a murine model of X-linked chronic granulomatous disorder. Methods in Molecular Biology, 1114, 427 - 440. doi:10.1007/978-1-62703-761-7-28
    • Chandrashekran A, Isa I, Dibb N, Winston R, Thrasher AJ, Casimir C, Readhead C, Dudhia J (2014). Lentiviral vector transduction of spermatozoa as a tool for the study of early development. FEBS Open Bio, 4, 266 - 275. doi:10.1016/j.fob.2014.02.008
    • Williams DA, Thrasher AJ (2014). Concise Review: Lessons Learned From Clinical Trials of Gene Therapy in Monogenic Immunodeficiency Diseases.. Stem Cells Transl Med, , - . doi:10.5966/sctm.2013-0206
    • Griffith LM, Cowan MJ, Puck JM, Dvorak CC, Notarangelo LD, Kohn DB, Pai S-Y, Ballard B, Boyle M, Hovermale E, Bauer SC, Bleesing JJH, Brower A, Buckley RH, Van Der Burg M, Burroughs LM, Candotti F, Cant AJ, Chatila T, Cunningham-Rundles C, Dinauer MC, Filipovich AH, Fleisher TA, Bobby Gaspar H, Gungor T, Haddad E, Huang F, Hurley A, Hurley M, Iyengar S, Kang EM, Malech HL, Logan BR, Long-Boyle JR, McGhee SA, Modell F, Modell V, Ochs HD, O'Reilly RJ, Parkman R, Rawlings DJ, Routes JM, Shearer WT, Small TN, Smith H, Sullivan KE, Szabolcs P, Thrasher A, Torgerson TR, Veys P, Weinberg K, Zuniga-Pflucker JC (2014). Primary Immune Deficiency Treatment Consortium (PIDTC) report. Journal of Allergy and Clinical Immunology, 133(2), - . doi:10.1016/j.jaci.2013.07.052
    • Mukherjee S, Thrasher AJ (2014). Gene correction of induced pluripotent stem cells derived from a murine model of x-linked chronic granulomatous disorder.. Methods Mol Biol, 1114, 427 - 440. doi:10.1007/978-1-62703-761-7_28
    • Griera M, Martin-Villar E, Banon-Rodríguez I, Blundell MP, Jones GE, Anton IM, Thrasher AJ, Rodriguez-Puyol M, Calle Y (2014). Integrin linked kinase (ILK) regulates podosome maturation and stability in dendritic cells.. Int J Biochem Cell Biol, , - . doi:10.1016/j.biocel.2014.01.021
    • Luevano M, Domogala A, Blundell M, Jackson N, Pedroza-Pacheco I, Derniame S, Escobedo-Cousin M, Querol S, Thrasher A, Madrigal A, Saudemont A (2014). Frozen cord blood hematopoietic stem cells differentiate into higher numbers of functional natural killer cells in vitro than mobilized hematopoietic stem cells or freshly isolated cord blood hematopoietic stem cells.. PLoS One, 9(1), e87086 - . doi:10.1371/journal.pone.0087086
    • Kane NM, Thrasher AJ, Angelini GD, Emanueli C (2014). MicroRNAs as modulators of stem cells and angiogenesis.. Stem Cells, , - . doi:10.1002/stem.1629
    • Bovellan M, Romeo Y, Biro M, Boden A, Chugh P, Yonis A, Vaghela M, Fritzsche M, Moulding D, Thorogate R, Jégou A, Thrasher AJ, Romet-Lemonne G, Roux PP, Paluch EK, Charras G (2014). Cellular control of cortical actin nucleation.. Curr Biol, 24(14), 1628 - 1635. doi:10.1016/j.cub.2014.05.069
    • Qasim W, Thrasher AJ (2014). Progress and prospects for engineered T cell therapies.. Br J Haematol, , - . doi:10.1111/bjh.12981
    • Van Til NP, Sarwari R, Visser TP, Van Der Velden G, Verstegen MMA, Wagemaker G, Hauer J, Lagresle-Peyrou C, Cavazzana M, Malshetty V, Cortes P, Jollet A, Danos O, Cassani B, Villa A, Zhang F, Thrasher AJ, Fontana E, Poliani PL (2014). Recombination-activating gene 1 (Rag1)-deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome. Journal of Allergy and Clinical Immunology, 133(4), 1116 - 1123. doi:10.1016/j.jaci.2013.10.009
    • Jonuschies J, Antoniou M, Waddington S, Boldrin L, Muntoni F, Thrasher A, Morgan J (2014). The Human Desmin Promoter Drives Robust Gene Expression for Skeletal Muscle Stem Cell-Mediated Gene Therapy.. Curr Gene Ther, , - .

    2013

    • Rivat C, Booth C, Alonso-Ferrero M, Blundell M, Sebire NJ, Thrasher AJ, Gaspar HB (2013). SAP gene transfer restores cellular and humoral immune function in a murine model of X-linked lymphoproliferative disease.. Blood, 121(7), 1073 - 1076. doi:10.1182/blood-2012-07-445858
    • Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D, Lochmüller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski J, Takeda S, Thrasher A, VandenDriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, Gao G (2013). Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.. Mol Ther, 21(2), 266 - 268. doi:10.1038/mt.2013.4
    • Booth C, Rivat C, Alonso-Ferrero M, Blundell M, Sebire NJ, Thrasher AJ, Gaspar HB (2013). Lentiviral-vector-mediated gene therapy for X-linked lymphoproliferative disease restores humoral and cellular functions.
    • Thrasher AJ (2013). Gene therapy for primary immunodeficiencies.
    • Leon-Rico D, Aldea M, Santilli G, Thrasher AJ, Bueren JA, Almarza E (2013). Development of new lentiviral vectors for the gene therapy of LAD-I.
    • Rothe M, Paul G, Schroeder S, Brendel C, Santilli G, Schambach A, Modlich U, Thrasher A, Grez M, Baum C (2013). In vivo gentoxicity profile of a chimeric promoter driven lentiviral vector for X-CGD in mice.
    • Syed F, Abdul-Wahab A, Georgiadis C, Petrova A, Ghani S, Thrasher A, McGrath J, Di W-L, Qasim W (2013). Lentiviral-mediated COL7A1 gene modified autologous cell therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB).
    • Carbonaro DA, Zhang L, Jin X, Montiel-Equihua C, Geiger S, Carmo M, Cooper A, Fairbanks L, Kaufman ML, Sebire NJ, Hollis RP, Blundell MP, Senadheera S, Fu PY, Sahaghian A, Chan RY, Wang X, Cornetta K, Thrasher AJ, Kohn DB, Gaspar HB (2013). Pre-clinical demonstration of lentiviral vector mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency.. Mol Ther, , - . doi:10.1038/mt.2013.265
    • Nowak K, Thrasher A, Di WL, Burns S (2013). The role of the common gamma chain in keratinocytes.
    • Beilin C, Choudhuri K, Bouma G, Malinova D, Llodra J, Stokes DL, Shimaoka M, Springer TA, Thrasher AJ, Dustin ML, Burns SO (2013). Dendritic cell-expressed common cytokine receptor g-chain is essential for effective IL-15 transpresentation to CD4+T cells at the immunological synapse.
    • Henriquez F, Schwoerer D, Gilmour K, Cale C, Thrasher AJ, Burns SO (2013). Functional apoptosis assays and serum biomarkers in the investigation of ALPS at great ormond street hospital.
    • Di WL, Mellerio JE, Bernadis C, Harper J, Abdul-Wahab A, Ghani S, Chan L, Martinez-Queipo M, Hara H, McNicol AM, Farzaneh F, McGrath J, Thrasher A, Qasim W (2013). Phase I study protocol for ex vivo lentiviral gene therapy for the inherited skin disease, netherton syndrome.. Hum Gene Ther Clin Dev, 24(4), 182 - 190. doi:10.1089/humc.2013.195
    • Zhan H, Gilmour K, Chan L, Farzaneh F, McNicol AM, Xu JH, Adams S, Fehse B, Veys P, Thrasher A, Gaspar H, Qasim W (2013). Production and First-in-Man Use of T Cells Engineered to Express a HSVTK-CD34 Sort-Suicide Gene.. PLoS One, 8(10), e77106 - . doi:10.1371/journal.pone.0077106
    • Zhang L, Thrasher AJ, Gaspar HB (2013). Current progress on gene therapy for primary immunodeficiencies. Gene Therapy, 20(10), 963 - 969. doi:10.1038/gt.2013.21
    • Di WL, Mellerio JE, Bernadis C, Harper J, Abdul-Wahab A, Ghani S, Martinez-Queipo M, Hara H, McNicol AM, McGrath J, Thrasher AJ, Qasim W (2013). Phase I study protocol for ex-vivo lentiviral gene therapy for the inherited skin disease, Netherton Syndrome.. Hum Gene Ther Clin Dev, , - . doi:10.1089/hum.2013.195
    • Moulding DA, Record J, Malinova D, Thrasher AJ (2013). Actin cytoskeletal defects in immunodeficiency.. Immunol Rev, 256(1), 282 - 299. doi:10.1111/imr.12114
    • Griffith LM, Cowan MJ, Puck JM, Dvorak CC, Notarangelo LD, Kohn DB, Pai S-Y, Ballard B, Boyle M, Hovermale E, Bauer SC, Bleesing JJH, Brower A, Buckley RH, van der Burg M, Burroughs LM, Candotti F, Cant AJ, Chatila T, Cunningham-Rundles C, Dinauer MC, Filipovich AH, Fleisher TA, Bobby Gaspar H, Gungor T, Haddad E, Huang F, Hurley A, Hurley M, Iyengar S, Kang EM, Malech HL, Logan BR, Long-Boyle JR, McGhee SA, Modell F, Modell V, Ochs HD, O'Reilly RJ, Parkman R, Rawlings DJ, Routes JM, Shearer WT, Small TN, Smith H, Sullivan KE, Szabolcs P, Thrasher A, Torgerson TR, Veys P, Weinberg K, Zuniga-Pflucker JC (2013). Primary Immune Deficiency Treatment Consortium (PIDTC) report. Journal of Allergy and Clinical Immunology, , - . doi:10.1016/j.jaci.2013.07.052
    • Jones RA, Feng Y, Worth AJ, Thrasher AJ, Burns SO, Martin P (2013). Modelling of human Wiskott-Aldrich syndrome protein mutants in zebrafish larvae using in vivo live imaging. JOURNAL OF CELL SCIENCE, 126(18), 4077 - 4084. doi:10.1242/jcs.128728
    • Choudhuri K, Beilin C, Bouma G, Llodra J, Malinova D, Stokes D, Springer T, Shimaoka M, Dustin M, Thrasher A, Burns S (2013). Dendritic cell-expressed common gamma-chain recruits IL-15 for trans-presentation at the immunological synapse.
    • Schachtner H, Calaminus SD, Sinclair A, Monypenny J, Blundell MP, Leon C, Holyoake TL, Thrasher AJ, Michie AM, Vukovic M, Gachet C, Jones GE, Thomas SG, Watson SP, Machesky LM (2013). Megakaryocytes assemble podosomes that degrade matrix and protrude through basement membrane.. Blood, 121(13), 2542 - 2552. doi:10.1182/blood-2012-07-443457
    • Charrier S, Cédrone G, Galy A, Blundell M, Thrasher AJ, Louache F, Vainchenker W (2013). Wiskott-Aldrich syndrome protein-deficient hematopoietic cells can be efficiently mobilized by granulocyte colony-stimulating factor. Haematologica, 98(8), 1300 - 1308. doi:10.3324/haematol.2012.077040
    • Santilli G, Anderson J, Thrasher AJ, Sala A (2013). Catechins and antitumor immunity: Not MDSC's cup of tea.. Oncoimmunology, 2(6), e24443 - . doi:10.4161/onci.24443
    • Georgiadis C, Semenova E, South AP, Thrasher A, McGrath JA, Di W-L, Qasim W (2013). Development of Lentiviral Gene Therapy for Type VII Collagen Deficient Epidermolysis Bullosa.
    • van Til NP, van der Velden G, de Boer H, Poliani PL, Antoniou MN, Villa A, Lagresle-Peyrou C, Cavazzana-Calvo M, Zhang F, Thrasher AJ, Wagemaker G (2013). Lentiviral Gene Therapy of RAG Severe Combined Immunodeficiency.
    • Pike-Overzet K, Wiekmeijer A-S, Baert MRM, Lagresle-Peyrou C, Schambach A, Zhang F, Hoeben RC, Hacein-Bey-Abina S, Lankester AC, Bredius RGM, Driessen GJA, Thrasher AJ, Baum C, Cavazzana-Calvo M, van Dongen JJM, Staal FJT (2013). Self-Inactivating Lentiviral Vectors for Correction of Rag1 Severe Combined Immunodeficiency.
    • Hacein-Bey-Abina S, Blondeau J, Caccavelli L, Charrier S, Picard C, Dal-Cortivo L, Cross G, Blanche S, Bushman F, Malani N, Fischer A, Thrasher A, Galy A, Cavazzana-Calvo M (2013). Lentiviral Vector-Based Gene Therapy for Wiskott-Aldrich Syndrome: Preliminary Results from the French Center.
    • Mukherjee S, Thrasher AJ (2013). Gene therapy for PIDs: Progress, pitfalls and prospects. Gene, 525(2), 174 - 181. doi:10.1016/j.gene.2013.03.098
    • Field A-C, Vink C, Gabriel R, Al-Subki R, Schmidt M, Goulden N, Stauss H, Thrasher A, Morris E, Qasim W (2013). Comparison of Lentiviral and Sleeping Beauty Mediated αβ T Cell Receptor Gene Transfer. PLoS ONE, 8(6), - .
    • Duncan G, Thrasher AJ, Kane NM (2013). Validation of haemogenic endothelial cell populations in the aorta gonad mesonephros of the mouse for microRNA analysis..
    • Kane N, Denby L, Duncan G, Mukherjee S, Blundell M, Baker A, Thrasher A (2013). MicroRNA profile of haemogenic endothelial cells and haematopoietic stem cells.
    • Knight S, Sanber K, Stephen S, Bailey R, Escors D, Santilli G, Thrasher A, Collins M, Takeuchi Y (2013). A clinical-grade constitutive packaging cell line for the production of self-inactivating lentiviral vectors.
    • Mukherjee S, Pipino C, David A, Blundell M, DeCoppi P, Thrasher A (2013). Trisomy 21 mid-trimester amniotic fluid iPS cells maintain genetic signatures during reprogramming: implications for disease modelling and cryo-banking.
    • Cheung W, Nowrouzi A, Coutelle C, Waddington S, Bigger B, Al Alaf F, Thrasher A, Andrews R, Wang Y, Montini E, Buckley S, Schmidt M, Reja S, Khonsari H, Bowden J, Newbold R, Roberts T, Themis M (2013). The fetal mouse is a model of genotoxicity that can uncover genes involved in liver cancer.
    • Moralli D, Mandegar M, Khoja S, Cowley S, Mukherjee S, Blundell M, Thrasher A, James W, Monaco Z (2013). Human artificial chromosomes maintained in human embryonic stem and neuronal cells.
    • Alsubki R, Field A-C, Hirwarkar P, Saudemont A, Stauss H, Morris E, Thrasher A, Pule M, Qasim W (2013). Engineering cord blood T cells for tumour immunotherapy.
    • Shangaris P, Loukogeorgakis SP, Bertin E, Maghsoudlou P, Blundell M, Urbani L, Ramachandra D, Spinelli V, Thrasher A, Pozzobon M, David AL, De Coppi P (2013). Amniotic fluid stem cells engraft the hematopoietic system following in utero transplantation.
    • Ghani S, Di W-L, Semenova E, Chan L, Mellerio J, McGrath J, Harper J, Thrasher A, Farzaneh F, Qasim W (2013). Production of gene modified epithelial sheets for phase 1 clinical trial for Netherton Syndrome.
    • Booth C, Rivat C, Ferrero MA, Blundell M, Sebire N, Thrasher A, Gaspar HB (2013). Lentiviral vector mediated gene therapy for X-linked lymphoproliferative disease (XLP) restores humoral and cellular functions.
    • Zhan H, Farzaneh F, Gilmour K, Chan L, McNicol A-M, Xu-Bayford JH, Adams S, Bhalla J, Swift S, Fehse B, Baum C, Veys P, Thrasher A, Gaspar HB, Qasim W (2013). First-in-man use of T cells engineered to express a HSVTK-CD34 sort-suicide gene.
    • Abdul-Razak HH, Rocca C, Howe SJ, Eugenia Alonso-Ferrero M, Gabriel R, Bartholomae CC, Gan CHV, Garin M, Javier Molina-Estevez F, Roberts AC, Blundell M, Gueenechea G, Holmes MC, Gregory PD, Kinnon C, von Kalle C, Schmidt M, Bueren JA, Thrasher AJ, Yanez-Munoz RJ (2013). Rescue of mouse Prkdc scid deficiency by transplantation of gene-repaired haematopoietic stem cells.
    • Qasim W, Brunetto M, Gehring A, Xue S-A, Zhan H, Ciccorossi P, Gilmour K, Swift S, Himoudi N, Cavallone D, Moriconi F, Farzhenah F, Mazzoni A, Chan L, Morris E, Thrasher A, Maini M, Bonino F, Staus H, Bertoletti A (2013). Production and use of autologous gene modified T cells targeting metastatic hepatitis B associated hepatocellular carcinoma.
    • Nowak K, Thrasher AJ, Di WL, Burns SO (2013). Keratinocytes express functional common gamma chain.
    • Di W, Ghani S, Almarza D, Chen I, Mellerio J, Bernadis C, Harper J, Thrasher A, Farzaneh F, Qasim W (2013). Production of gene engineered epithelia sheets for first-in-man use.
    • Zhang L, Thrasher AJ, Gaspar HB (2013). Current progress on gene therapy for primary immunodeficiencies.. Gene Ther, , - . doi:10.1038/gt.2013.21
    • Moeendarbary E, Valon L, Fritzsche M, Harris AR, Charras GT, Moulding DA, Thrasher AJ, Stride E, Mahadevan L (2013). The cytoplasm of living cells behaves as a poroelastic material. Nature Materials, 12(3), 253 - 261. doi:10.1038/nmat3517
    • Lang PA, Brenner D, Mak TW, Ohashi PS, Shaabani N, Honke N, Meryk A, Häussinger D, Lang KS, Borkens S, Scheu S, Barthuber C, Booth S, Griffiths GM, Recher M, Thrasher AJ, Bouma G (2013). Reduced type I interferon production by dendritic cells and weakened antiviral immunity in patients with Wiskott-Aldrich syndrome protein deficiency. Journal of Allergy and Clinical Immunology, 131(3), - . doi:10.1016/j.jaci.2012.08.050
    • Morris E, Gore M, Baker A, Thrasher AJ (2013). Moving forward on shifting sands: ethical regulation of gene therapy clinical trials in the United kingdom.. Mol Ther, 21(4), 715 - 716. doi:10.1038/mt.2013.43
    • Thrasher AJ (2013). Progress in lentiviral vector technologies. Human Gene Therapy, 24(2), 117 - 118. doi:10.1089/hum.2013.2500
    • Nowrouzi A, Cheung WT, Li T, Zhang X, Arens A, Paruzynski A, Waddington SN, Osejindu E, Reja S, von Kalle C, Wang Y, Al-Allaf F, Gregory L, Themis M, Holder M, Dighe N, Ruthe A, Buckley SM, Bigger B, Montini E, Thrasher AJ, Andrews R, Roberts TP, Newbold RF, Coutelle C, Schmidt M, Themis M (2013). The fetal mouse is a sensitive genotoxicity model that exposes lentiviral-associated mutagenesis resulting in liver oncogenesis.. Mol Ther, 21(2), 324 - 337. doi:10.1038/mt.2012.224
    • Moschidou D, Mukherjee S, Blundell MP, Jones GN, Atala AJ, Thrasher AJ, Fisk NM, De Coppi P, Guillot PV (2013). Human mid-trimester amniotic fluid stem cells cultured under embryonic stem cell conditions with valproic acid acquire pluripotent characteristics.. Stem Cells Dev, 22(3), 444 - 458. doi:10.1089/scd.2012.0267
    • Gaspar HB, Swift S, Thrasher AJ (2013). "Special exemptions": should they be put on trial?. Mol Ther, 21(2), 261 - 262. doi:10.1038/mt.2013.1
    • Santilli G, Piotrowska I, Cantilena S, Chayka O, D'Alicarnasso M, Morgenstern DA, Himoudi N, Pearson K, Anderson J, Thrasher AJ, Sala A (2013). Polyphenon [corrected] E enhances the antitumor immune response in neuroblastoma by inactivating myeloid suppressor cells.. Clin Cancer Res, 19(5), 1116 - 1125. doi:10.1158/1078-0432.CCR-12-2528
    • Furmanski AL, O'Shaughnessy RF, Saldana JI, Blundell MP, Thrasher AJ, Sebire NJ, Davies EG, Crompton T (2013). T-cell reconstitution after thymus xenotransplantation induces hair depigmentation and loss.. J Invest Dermatol, 133(5), 1221 - 1230. doi:10.1038/jid.2012.492
    • Moeendarbary E, Valon L, Fritzsche M, Harris AR, Moulding DA, Thrasher AJ, Stride E, Mahadevan L, Charras GT (2013). The cytoplasm of living cells behaves as a poroelastic material.. Nat Mater, 12(3), 253 - 261. doi:10.1038/nmat3517
    • Rocca C, Abdul-Razak HH, Howe SJ, Alonso-Ferrero ME, Gabriel R, Bartholomae CC, Gan CHV, Garin M, Molina Estevez FJ, Roberts A, Blundell M, Wang J, Prakash V, Gueenechea G, Holmes MC, Gregory PD, Kinnon C, von Kalle C, Schmidt M, Antonio Bueren J, Thrasher AJ, Yanez-Munoz RJ (2013). Rescue of T-cell deficiency in PRKDC SCID mice by transplantation of gene-edited haematopoietic stem cells.
    • Chan E, Mussolino C, Thrasher A, Cathomen T, Towers G, Qasim W (2013). Mimicry of anti-HIV retrotransposition events by TALEN mediated Cyclophilin insertion into the human TRIM5 locus.
    • van Til NP, Sarwari R, Visser TP, van der Velden G, Verstegen MMA, Wagemaker G, Hauer J, Lagresle-Peyrou C, Cavazzana M, Malshetty V, Cortes P, Jollet A, Danos O, Cassani B, Villa A, Zhang F, Thrasher AJ, Fontana E, Poliani PL (2013). Recombination-activating gene 1 (Rag1)-deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome. Journal of Allergy and Clinical Immunology, , - . doi:10.1016/j.jaci.2013.10.009
    • Ginn SL, Liao SHY, Hallwirth CV, Teber ET, Reddel RR, McCormack M, Thrasher AJ, Hu M, Alexander SI, Alexander IE (2013). STRESS HAEMATOPOIESIS AND MALIGNANCY: IMPLICATIONS FOR EMERGING GENE AND CELL-BASED THERAPIES.
    • Bardelli M, Mukherjee S, Tai A, Linden M, Thrasher A, Henckaerts E (2013). Patient-specific induced pluripotent stem (iPS) cells as a model to study feasibility and safety of AAV-mediated targeted gene addition.
    • Carmo M, Arumugam P, Montiel-Equihua CA, Alonso-Ferrero ME, Schambach A, Baum C, Risma K, Malik P, Thrasher AJ, Jordan MB, Gaspar B (2013). Development of gene therapy for hemophagocytic lymphohistiocytosis (HLH) due to perforin deficiency - investigation of whether expression of perforin in stem cells has a toxic effect.
    • Mukherjee S, Thrasher AJ (2013). Gene therapy for PIDs: progress, pitfalls and prospects.. Gene, 525(2), 174 - 181. doi:10.1016/j.gene.2013.03.098
    • Boucherie C, Mukherjee S, Henckaerts E, Thrasher AJ, Sowden JC, Ali RR (2013). Brief report: self-organizing neuroepithelium from human pluripotent stem cells facilitates derivation of photoreceptors.. Stem Cells, 31(2), 408 - 414. doi:10.1002/stem.1268

    2012

    • Cole TS, Jones LKR, McGrogan P, Pearce MS, Flood TJ, Cant AJ, Goldblatt D, Thrasher AJ, Gennery AR, McKendrick F, Titman P (2012). Emotional and behavioural difficulties in chronic granulomatous disease. ARCHIVES OF DISEASE IN CHILDHOOD, 97(1), 87 - 87. doi:10.1136/archdischild-2011-300780
    • Kallinikou K, Linch DC, Yong KL, Anjos-Afonso F, Bonnet D, Blundell MP, Thrasher AJ, Ings SJ, Watts MJ (2012). Engraftment defect of cytokine-cultured adult human mobilized CD34 cells is related to reduced adhesion to bone marrow niche elements. British Journal of Haematology, 158(6), 778 - 787. doi:10.1111/j.1365-2141.2012.09219.x
    • Gaspar B, Carmo M, Arumugam P, Alonso-Ferrero M, Schambach A, Baum C, Risma K, Malik P, Jordan M, Rivat C, Booth C, Thrasher A (2012). Development of Gene therapy for HLH due to perforin deficiency and for XLP1.
    • Sung P, Mukherjee S, Thrasher AJ (2012). Lineage-specific haematopoietic differentiation of human induced pluripotent stem cells.
    • Leon-Rico D, Aldea M, Sanchez R, Segovia JC, Santilli G, Thrasher AJ, Bueren JA, Almarza E (2012). Itgb2 hypomorphic mice: a novel approach for ex vivo LAD gene therapy.
    • Burns SO, Thrasher AJ, Zenner HL, Curtis J, Nejentsev S, Plagnol V, Mok K, Eisenhut M, Kumararatne D, Doffinger R (2012). LRBA gene deletion in a patient presenting with autoimmunity without hypogammaglobulinemia. Journal of Allergy and Clinical Immunology, 130(6), 1428 - 1432. doi:10.1016/j.jaci.2012.07.035
    • Guasti L, Prasongchean W, Kleftorious G, Mukherjee S, Thrasher AJ, Bulstrode NW, Ferretti P (2012). High plasticity of paediatric adipose tissue-derived stem cells: too much for selective skeletogenic differentiation?. Stem Cells Trans Med, 1(5), 384 - 395. doi:10.5966/sctm.2012-0009
    • Carmo M, Arumugam P, Montiel-Equiha C, Alonso-Ferrero M, Schambach A, Baum C, Risma K, Malik P, Thrasher A, Jordan M, Gaspar B (2012). DEVELOPMENT OF GENE THERAPY FOR HAEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS (HLH) DUE TO PERFORIN DEFICIENCY.
    • Booth C, Rivat C, Blundell M, Alonso-Ferrera M, Thrasher A, Gaspar B (2012). LENTIVIRAL VECTOR MEDIATED GENE THERAPY FOR X-LINKED LYMPHOPROLIFERATIVE DISEASE RESTORES HUMORAL AND CELLULAR FUNCTIONS.
    • Chiew M-L, Ballard S, Stauss H, Thrasher A, Longhurst H, Workman S (2012). WORLD IMMUNOLOGY DAY 2012 EDUCATIONAL SYMPOSIUM FOR CLINICIANS AND PATIENTS.
    • Bouma G, Carter NA, Recher M, Notarangelo LD, Burns SO, Mauri C, Thrasher AJ (2012). DEFICIENCY OF REGULATORY B CELLS IN WISKOTT-ALDRICH SYNDROME PROTEIN DEFICIENT MICE LEADS TO EXACERBATED AUTOIMMUNE ARTHRITIS.
    • Burns S, Zenner H, Plagnol V, Curtis J, Mok K, Eisenhut M, Kumararatne D, Doffinger R, Thrasher A, Nejentsev S (2012). EXOME SEQUENCINGIDENTIFIES LRBA GENE DELETION IN A PATIENT PRESENTING WITH AUTOIMMUNITY WITHOUT HYPOGAMMAGLOBULINEMIA.
    • Worth A, Metelo J, Bouma G, Vernay B, Fritzsche M, Charras G, Thrasher A, Burns S (2012). DISEASE ASSOCIATED MISSENSE MUTATIONS IN THE EVH1 DOMAIN DISRUPT INTRINSIC WASP FUNCTION CAUSING DYSREGULATED ACTIN DYNAMICS AND IMPAIRED DENDRITIC CELL MIGRATION.
    • Bouma G, Lang PA, Shaabani N, Borkens S, Scheu S, Booth S, Honke N, Brenner D, Meryk A, Recher M, Mak TW, Ohashi PS, Hausinger D, Griffiths GM, Thrasher AJ, Lang KS (2012). REDUCED TYPE I INTERFERON PRODUCTION BY DENDRITIC CELLS AND WEAKENED ANTIVIRAL IMMUNITY IN WISKOTT-ALDRICH SYNDROME PROTEIN DEFICIENCY.
    • Davies EG, Gilmour KC, Parsley K, Curry J, Sebire N, Poliani L, McCarthy EA, Devlin B, Markert ML, Thrasher AJ (2012). THYMUS TRANSPLANTATION FOR COMPLETE DIGEORGE SYNDROME: THE LONDON EXPERIENCE.
    • Georgiadis C, Thrasher A, South AP, McGrath J, Qasim W, Di W (2012). Development of lentiviral gene therapy for type VII collagen deficient epidermolysis bullosa.
    • Plagnol V, Curtis J, Epstein M, Mok KY, Stebbings E, Grigoriadou S, Wood NW, Hambleton S, Burns SO, Thrasher AJ, Kumararatne D, Doffinger R, Nejentsev S (2012). A robust model for read count data in exome sequencing experiments and implications for copy number variant calling.. Bioinformatics, 28(21), 2747 - 2754. doi:10.1093/bioinformatics/bts526
    • Pike-Overzet K, Rodijk M, Ng YY, Baert M, Lagresle-Peyrou C, Schambach A, Zhang F, Hoeben R, Hacein-Bey-Abina S, Lankester A, Bredius R, Driessen G, Thrasher A, Baum C, Cavazzana-Calvo M, van Dongen J, Staal F (2012). SELF-INACTIVATING LENTIVIRAL VECTORS FOR CORRECTION OF RAG1 SEVERE COMBINED IMMUNODEFICIENCY.
    • Moschidou D, Mukherjee S, Blundell MP, Drews K, Jones GN, Abdulrazzak H, Nowakowska B, Phoolchund A, Lay K, Ramasamy TS, Cananzi M, Nettersheim D, Sullivan M, Frost J, Moore G, Vermeesch JR, Fisk NM, Thrasher AJ, Atala A, Adjaye J, Schorle H, De Coppi P, Guillot PV (2012). Valproic acid confers functional pluripotency to human amniotic fluid stem cells in a transgene-free approach.. Mol Ther, 20(10), 1953 - 1967. doi:10.1038/mt.2012.117
    • Lowdell MW, Birchall M, Thrasher AJ (2012). Use of compassionate-case ATMP in preclinical data for clinical trial applications.. Lancet, 379(9834), 2341 - . doi:10.1016/S0140-6736(12)61018-6
    • Knight S, Zhang F, Mueller-Kuller U, Bokhoven M, Gupta A, Broughton T, Sha S, Antoniou MN, Brendel C, Grez M, Thrasher AJ, Collins M, Takeuchi Y (2012). Safer, silencing-resistant lentiviral vectors: optimization of the ubiquitous chromatin-opening element through elimination of aberrant splicing.. J Virol, 86(17), 9088 - 9095. doi:10.1128/JVI.00485-12
    • Rivat C, Santilli G, Gaspar HB, Thrasher AJ (2012). Gene therapy for primary immunodeficiencies.. Hum Gene Ther, 23(7), 668 - 675. doi:10.1089/hum.2012.116
    • Mohanty ST, Cairney CJ, Chantry AD, Madan S, Fernandes JA, Howe SJ, Moore HD, Thompson MJ, Chen B, Thrasher A, Keith WN, Bellantuono I (2012). A Small Molecule Modulator of Prion Protein Increases Human Mesenchymal Stem Cell Lifespan, Ex Vivo Expansion, and Engraftment to Bone Marrow in NOD/SCID Mice. STEM CELLS, 30(6), 1134 - 1143. doi:10.1002/stem.1065
    • Bardelli M, Zhang F, Linden RM, Thrasher AJ, Henckaerts E (2012). AAV-Mediated Targeted Gene Addition in Patient-Specific SCID-X1 Induced Pluripotent Stem Cells.
    • Shaw SWS, David AL, Blundell M, Howe S, Pipino C, Maghsoudlou P, Lin J, Porada CD, Thrasher A, De Coppi P (2012). CD34+cells derived from sheep amniotic fluid could engraft in NOD-SCID gamma mice and in lambs after prenatal autologous transplantation.
    • Ward N, Buckley S, Rahim A, McVey J, Thrasher A, Waddington S (2012). Codon optimised FVIII constructs for treatment of haemophilia A.
    • van Til NP, de Boer H, Huston M, Fontana E, Poliani PL, Cassani B, Antoniou MN, Zhang F, Thrasher AJ, Villa A, Wagemaker G (2012). Correction of Immunity in Murine Rag2 Severe Combined Immunodeficiency by Lentiviral Gene Therapy Using a Codon-Optimized RAG2 Therapeutic Transgene.
    • Montiel-Equihua CA, Zhang L, Knight S, Saadeh H, Scholz S, Carmo M, Alonso-Ferrero ME, Blundell MP, Monkeviciute A, Schulz R, Collins M, Takeuchi Y, Schmidt M, Fairbanks L, Antoniou M, Thrasher AJ, Gaspar HB (2012). Development of a gene expression system for enhanced erythroid expression.
    • Carbonaro DA, Jin X, Geiger S, Senadheera S, Montiel-Equihua C, Gaspar B, Thrasher A, Hollis RP, Kohn DB (2012). Development of a Lentiviral Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immunodeficiency (ADA-SCID).
    • Carmo M, Arumugam P, Montiel-Equihua CA, Alonso-Ferrero ME, Tiwari S, Schambach A, Baum C, Risma K, Malik P, Thrasher AJ, Jordan MB, Gaspar B (2012). Development of Gene Therapy for Hemophagocytic Lymphohistiocytosis (HLH) Due to Perforin Deficiency.
    • Carmo M, Arumugam P, Montiel-Equihua CA, Alonso-Ferrero ME, Schambach A, Baum C, Risma K, Malik P, Thrasher AJ, Jordan MB, Gaspar B (2012). Development of gene therapy for hemophagocytic lymphohistiocytosis (HLH) due to perforin deficiency.
    • Alonso-Ferrero ME, Langford-Smith A, Burke D, Heales S, Bigger BW, Thrasher A, Gaspar B (2012). Gene Therapy for Lysosomal Storage Diseases using an enhanced lentiviral vector delivery system.
    • Zhang F, Knight S, Collins M, Takeuchi Y, Antoniou M, Thrasher AJ (2012). Improved safety of Lentiviral vectors containing silencing resistant A2UCOE regulatory elements.
    • Mukherjee S, Pipino C, Sung P, DeCoppi P, Thrasher A (2012). Investigating perturbed haematopoiesis in Down Syndrome patients: an induced pluripotent stem cell approach.
    • Chan E, Schaller T, Tan CP, Thrasher AJ, Towers GJ, Qasim W (2012). Lentiviral Gene Therapy Against HIV-1 Using a Novel TRIM21-Cyclophilin A Fusion Restriction Factor.
    • Acosta-Saltos C, Gonitel R, Acosta-Saltos A, Chavda S, Thrasher A, Anderson P, Raivich G (2012). Lentiviral GMCSF is neuroinflammatory and proregenerative.
    • Rivat C, Booth C, Blundell M, Thrasher AJ, Gaspar HB (2012). Lentiviral vector mediated gene therapy for X-linked lymphoproliferative disease (XLP1) restores humoral and cellular functions.
    • Rivat C, Booth C, Blundell M, Thrasher AJ, Gaspar HB (2012). Lentiviral Vector Mediated Gene Therapy for X-Linked Lymphoproliferative Disease Restores Humoral and Cellular Functions.
    • Pantoglou J, Abdul-Razak HH, Rocca C, Howe SJ, Gregory PD, Holmes MC, Kinnon C, Thrasher AJ, Yanez-Munoz RJ (2012). Specificity of a zinc-finger nuclease targeting mouse Prkdc: preparation of samples for analysis of off-target cutting.
    • Pallant C, Cathomen T, Dannemann N, Qasim W, Naldini L, Kinnon C, Howe S, Thrasher A (2012). Targeting models of severe combined immunodeficiency for correction by zinc finger nucleases.
    • Semenova E, Ghani S, Chan L, Farzaneh F, Larcher F, Harper J, Thrasher A, Qasim W, Di WL (2012). Clinical grade lentiviral vector production for inherited skin disease.
    • Montiel-Equihua CA, Zhang L, Knight S, Saadeh H, Scholz S, Carmo M, Alonso-Ferrero ME, Blundell MP, Monkeviciute A, Schulz R, Collins M, Takeuchi Y, Schmidt M, Fairbanks L, Antoniou M, Thrasher AJ, Gaspar HB (2012). The β-globin locus control region in combination with the EF1α short promoter allows enhanced lentiviral vector-mediated erythroid gene expression with conserved multilineage activity.. Mol Ther, 20(7), 1400 - 1409. doi:10.1038/mt.2012.50
    • Jonuschies J, Boldrin L, Thrasher A, Muntoni F, Morgan J (2012). Assessment of potential promoters for lentiviral gene therapy in DMD.
    • Recher M, Burns SO, de la Fuente MA, Volpi S, Dahlberg C, Walter JE, Moffitt K, Mathew D, Honke N, Lang PA, Patrizi L, Falet H, Keszei M, Mizui M, Csizmadia E, Candotti F, Nadeau K, Bouma G, Delmonte OM, Frugoni F, Fomin AB, Buchbinder D, Lundequist EM, Massaad MJ, Tsokos GC, Hartwig J, Manis J, Terhorst C, Geha RS, Snapper S, Lang KS, Malley R, Westerberg L, Thrasher AJ, Notarangelo LD (2012). B cell-intrinsic deficiency of the Wiskott-Aldrich syndrome protein (WASp) causes severe abnormalities of the peripheral B-cell compartment in mice.. Blood, 119(12), 2819 - 2828. doi:10.1182/blood-2011-09-379412
    • Montiel-Equihua CA, Thrasher AJ, Gaspar HB (2012). Gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency.. Curr Gene Ther, 12(1), 57 - 65.
    • Tolar J, Becker PS, Clapp DW, Hanenberg H, Diaz de Heredia C, Kiem H-P, Navarro S, Qasba P, Rio P, Schmidt M, Sevilla J, Verhoeyen E, Thrasher AJ, Bueren J (2012). Gene Therapy for Fanconi Anemia: One Step Closer to the Clinic. HUMAN GENE THERAPY, 23(2), 141 - 144. doi:10.1089/hum.2011.237
    • Carmo M, Arumugam P, Tiwari S, Thrasher A, Risma K, Malik P, Gaspar B, Jordan M (2012). GENE THERAPY FOR HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS (HLH): FIXING A CRITICAL 'CIRCUIT BREAKER' IN THE IMMUNE SYSTEM.
    • Van Til NP, De Boer H, Mashamba N, Wabik A, Huston M, Visser TP, Wagemaker G, Fontana E, Poliani PL, Cassani B, Zhang F, Thrasher AJ, Villa A (2012). Correction of murine rag2 severe combined immunodeficiency by lentiviral gene therapy using a codon-optimized RAG2 therapeutic transgene. Molecular Therapy, 20(10), 1968 - 1980. doi:10.1038/mt.2012.110
    • Seymour LW, Thrasher AJ (2012). Gene therapy matures in the clinic. Nature Biotechnology, 30(7), 588 - 593. doi:10.1038/nbt.2290
    • Himoudi N, Wallace R, K P, Poon E, Gilmour K, A B, Howe K, Dong R, Sebire N, Michalski A, Thrasher A, Anderson J (2012). Lack of T cell responses following autologous tumor lysate pulsed dendritic cell vaccination, in patients with relapsed osteosarcoma.. Clinical and Translational Oncology, 14(4), 271 - 279.
    • Burns SO, Zenner HL, Plagnol V, Curtis J, Mok K, Eisenhut M, Kumararatne D, Doffinger R, Thrasher AJ, Nejentsev S (2012). LRBA gene deletion in a patient presenting with autoimmunity without hypogammaglobulinemia. Journal of Allergy and Clinical Immunology, , - .
    • Cooray S, Howe SJ, Thrasher AJ (2012). Retrovirus and lentivirus vector design and methods of cell conditioning.. Methods Enzymol, 507, 29 - 57. doi:10.1016/B978-0-12-386509-0.00003-X
    • Van Der Loo JCM, Grassman E, Terwilliger A, Higashimoto T, Nordling DL, Malik P, Swaney WP, Schambach A, Baum C, Thrasher AJ, Williams DA, Reeves L (2012). Scale-up and manufacturing of clinical-grade self-inactivating γ-retroviral vectors by transient transfection. Gene Therapy, 19(3), 246 - 254. doi:10.1038/gt.2011.102
    • Montiel-Equihua CA, Zhang L, Carmo M, Alonso-Ferrero ME, Blundell MP, Monkeviciute A, Thrasher AJ, Bobby Gaspar H, Knight S, Collins M, Saadeh H, Schulz R, Antoniou M, Scholz S, Schmidt M, Takeuchi Y, Fairbanks L (2012). The Β-globin locus control region in combination with the EF1α short promoter allows enhanced lentiviral vector-mediated erythroid gene expression with conserved multilineage activity. Molecular Therapy, 20(7), 1400 - 1409. doi:10.1038/mt.2012.50
    • Moschidou D, Jones GN, Abdulrazzak H, Phoolchund A, Lay K, Ramasamy TS, Sullivan M, Guillot PV, Mukherjee S, Blundell MP, Cananzi M, Frost J, Moore G, Thrasher AJ, De Coppi P, Drews K, Adjaye J, Nowakowska B, Vermeesch JR, Nettersheim D, Schorle H, Fisk NM, Atala A (2012). Valproic acid confers functional pluripotency to human amniotic fluid stem cells in a transgene-free approach. Molecular Therapy, 20(10), 1953 - 1967. doi:10.1038/mt.2012.117
    • Macpherson L, Monypenny J, Blundell MP, Cory GO, Tomé-García J, Thrasher AJ, Jones GE, Calle Y (2012). Tyrosine phosphorylation of WASP promotes calpain-mediated podosome disassembly.. Haematologica, 97(5), 687 - 691. doi:10.3324/haematol.2011.048868
    • Di WL, Semenova E, Larcher F, Del Rio M, Harper JI, Thrasher AJ, Qasim W (2012). Human involucrin promoter mediates repression-resistant and compartment-specific LEKTI expression.. Hum Gene Ther, 23(1), 83 - 90. doi:10.1089/hum.2011.091
    • Moeendarbary E, Valon L, Harris A, Moulding D, Thrasher A, Stride E, Mahadevan L, Charras G (2012). Cellular Rheology and Hydraulics. BIOPHYSICAL JOURNAL, 102(3), 563A - 563A.
    • Worth AJJ, Metelo J, Bouma G, Moulding D, Fritzsche M, Vernay B, Charras G, Cory GOC, Thrasher AJ, Burns SO (2012). Disease-associated missense mutations in the EVH1 domain disrupt intrinsic WASp function causing dysregulated actin dynamics and impaired dendritic cell migration. Blood, In press, - .
    • Chick HE, McDonald RA, Kane NM, Alba R, Delles C, Baker AH, Nowrouzi A, Fronza R, Schmidt M, Thrasher AJ, Sessa WC (2012). Integrase-deficient lentiviral vectors mediate efficient gene transfer to human vascular smooth muscle cells with minimal genotoxic risk. Human Gene Therapy, 23(12), 1247 - 1257. doi:10.1089/hum.2012.042
    • Pike-Overzet K, Rodijk M, Ng YY, Baert MRM, Lagresle-Peyrou C, Schambach A, Zhang F, Hoeben RC, Hacein-Bey-Abina S, Lankester AC, Bredius RGM, Driessen GJA, Thrasher AJ, Baum C, Cavazzana-Calvo M, van Dongen JJM, Staal FJT (2012). Self-Inactivating Lentiviral Vectors for Correction of Rag1 Severe Combined Immunodeficiency.
    • Chan E, Schaller T, Eddaoudi A, Zhan H, Tan CP, Jacobsen M, Thrasher AJ, Towers GJ, Qasim W (2012). Lentiviral Gene Therapy Against Human Immunodeficiency Virus Type 1, Using a Novel Human TRIM21-Cyclophilin A Restriction Factor. HUMAN GENE THERAPY, 23(11), 1176 - 1185. doi:10.1089/hum.2012.083
    • Charrier S, Blundell M, Cedrone G, Louache F, Vainchenker W, Thrasher A, Galy A (2012). Efficient mobilization and transduction of WASP-deficient haematopoietic cells in a murine model.
    • van der Loo JCM, Swaney WP, Grassman E, Terwilliger A, Higashimoto T, Schambach A, Hacein-Bey-Abina S, Nordling DL, Cavazzana-Calvoo M, Thrasher AJ, Williams DA, Reeves L, Malik P (2012). Critical Variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency. Gene Therapy, , - .
    • Plagnol V, Curtis J, Epstein M, Mok K, Stebbings E, Grigoriadou S, Wood N, Hambleton S, Burns S, Thrasher A, Kumararatne D, Doffinger R, Nejentsev S (2012). NEW POWERFUL SOFTWARE FOR COPY NUMBER ANALYSIS OF EXOME SEQUENCE DATA, EXOMEDEPTH, DISCOVERS NOVEL MUTATIONS IN PRIMARY IMMUNODEFICIENCY PATIENTS.
    • Abdul-Razak HH, Rocca C, Howe SJ, Estevez JFM, Gan CHV, Roberts A, Gregory PD, Holmes MC, Kinnon C, Thrasher AJ, Yanez-Munoz RJ (2012). Correction of mouse Prkdc scid deficiency by ZFN-driven gene targeting.
    • Van Der Loo JCM, Grassman E, Terwilliger A, Higashimoto T, Nordling DL, Malik P, Swaney WP, Schambach A, Hacein-Bey-Abina S, Cavazzana-Calvo M, Thrasher AJ, Williams DA, Reeves L (2012). Critical Variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency. Gene Therapy, 19(8), 872 - 876. doi:10.1038/gt.2012.37

    2011

    • Binny C, McIntosh J, Peruta M, Kymalainen H, Tuddenham E, Waddington S, Buckley S, Spence Y, Morton C, Thrasher A, Gray J, Tarantal A, Davidoff A, Nathwani A (2011). AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous haemorrhage. HUMAN GENE THERAPY, 22(10), A45 - A45.
    • Pike-Overzet K, Rodijk M, Ng YY, Baert MRM, Lagresle-Peyrou C, Schambach A, Zhang F, Hoeben RC, Hacein-Bey-Abina S, Lankester AC, Bredius RGM, Driessen GJA, Thrasher AJ, Baum C, Cavazzana-Calvo M, van Dongen JJM, Staal FJT (2011). Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer. LEUKEMIA, 25(9), 1471 - 1483. doi:10.1038/leu.2011.106
    • Beilin C, Choudhuri K, Bouma G, Thrasher AJ, Dustin ML, Burns SO (2011). Dendritic cell common G-chain cytokine receptor is required for optimal IL-15 transpresentation to CD4+T cells at the immunological synapse.
    • Booth C, Gaspar HB, Thrasher AJ (2011). Gene therapy for primary immunodeficiency.. Curr Opin Pediatr, 23(6), 659 - 666. doi:10.1097/MOP.0b013e32834cd67a
    • Kallinikou K, Anjos-Afonso F, Blundell MP, Ings SJ, Kassen D, Thrasher AJ, Linch DC, Bonnet D, Yong K (2011). CD26 Inhibition Can Aid the Homing of Cytokine Activated Mobilized Peripheral Blood (MPB)CD34+Cells to the Bone Marrow (BM) but a Ligand Dependent Attachment Defect Prevents Their Long Term Retention and Subsequent Engraftment.
    • Deichmann A, Brugman MH, Bartholomae CC, Schwarzwaelder K, Verstegen MMA, Howe SJ, Arens A, Ott MG, Hoelzer D, Seger R, Grez M, Hacein-Bey-Abina S, Cavazzana-Calvo M, Fischer A, Paruzynski A, Gabriel R, Glimm H, Abel U, Cattoglio C, Mavilio F, Cassani B, Aiuti A, Dunbar CE, Baum C, Gaspar HB, Thrasher AJ, von Kalle C, Schmidt M, Wagemaker G (2011). Insertion Sites in Engrafted Cells Cluster Within a Limited Repertoire of Genomic Areas After Gammaretroviral Vector Gene Therapy. MOL THER, 19(11), 2031 - 2039. doi:10.1038/mt.2011.178
    • Galy A, Thrasher AJ (2011). Gene therapy for the Wiskott-Aldrich syndrome.. Curr Opin Allergy Clin Immunol, 11(6), 545 - 550. doi:10.1097/ACI.0b013e32834c230c
    • Jonuschies J, Boldrin L, Thrasher A, Morgan JE, Muntoni F (2011). Assessment of potential promoters for lentiviral gene therapy in DMD. HUM GENE THER, 22(10), A62 - A63.
    • Jonuschies J, Boldrin L, Thrasher A, Morgan JE, Muntoni F (2011). Assessment of potential promoters for lentiviral gene therapy in DMD. HUM GENE THER, 22(10), A5 - A5.
    • Farmer S, Lorain S, Thrasher A, Garcia L, Muntoni F, Conti F (2011). Correction of FKRP function via RNA trans-splicing. doi:10.1016/j.nmd.2011.06.1016
    • Kao VYC, Waddington S, Equihua CM, Thrasher AJ, Antoniou MN (2011). Curative F.IX production from low-dose UCOE-lentiviral vectors. HUM GENE THER, 22(10), A110 - A110.
    • Montiel-Equihua CA, Zhang L, Knight S, Collins M, Saadeh H, Schulz R, Scholz S, Schmidt M, Carmo M, Alonso-Ferrero ME, Blundell MP, Fairbanks LD, Monkeviciute A, Antoniou M, Thrasher AJ, Gaspar HB (2011). Development of a gene expression system for enhanced erythroid expression. HUMAN GENE THERAPY, 22(10), A105 - A105.
    • Carmo M, Montiel-Equihua CA, Schambach A, Baum C, Thrasher AJ, Gaspar HB (2011). Development of gene therapy for haemophagocytic lymphohistiocytosis (HLH) due to perforin deficiency. HUM GENE THER, 22(10), A46 - A47.
    • Zhang L, Montiel-Equihua CA, Thrasher AJ, Gaspar HB (2011). Dr Lin Zhang. HUMAN GENE THERAPY, 22(10), A53 - A54.
    • Hacein-Bey-Abina S, Caccaveli L, Touzot F, Dal-Cortivo L, Heritier S, Frange P, Thrasher A, Gaspar B, Baum C, Schambach A, Malik P, Van der Loo H, Williams D, Bushman F, de Saint-Basile G, Lim A, Debre M, Blanche S, Fischer A, Cavazzana-Calvo M (2011). Efficacy of gene therapy for X-linked severe combined immunodeficiency: update on trial No.1 follow-up and preliminary results of multicentric collaborative trial No. 2 (Paris - Boston - London). HUMAN GENE THERAPY, 22(10), A40 - A40.
    • Thrasher A (2011). Gene therapy for immunodeficiency. HUMAN GENE THERAPY, 22(10), A5 - A6.
    • Alonso-Ferrero ME, Burke D, Heales S, Thrasher A, Gaspar HB (2011). Gene therapy for lysosomal storage diseases using an enhanced lentiviral vector delivery system. HUM GENE THER, 22(10), A44 - A45.
    • Cooray S, Gilmour KC, Parsley KL, Zhang F, Bjorkegren E, Bayford J, Brown L, Davies EG, Veys P, Fairbanks L, Bordon V, Petropolou T, Kinnon C, Thrasher AJ, Gaspar HB (2011). Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction. HUM GENE THER, 22(10), A40 - A40.
    • Semenova E, Di WL, Larcher F, Ghani S, Harper J, Thrasher A, Qasim W (2011). Human involucrin promoter resists spreading methylation and supports compartment specific gene expression in engineered skin grafts. HUM GENE THER, 22(10), A52 - A52.
    • Chan E, Schaller T, Thrasher A, Towers G, Qasim W (2011). Lentivirus mediated protection against HIV-1 using a human TRIM21-Cyclophilin A fusion protein. HUM GENE THER, 22(10), A68 - A68.
    • Ward NJ, Buckley SMK, Rahim AA, McVey JH, Thrasher AJ, Waddington SN (2011). Optimised vectors for the treatment of haemophilia A. HUM GENE THER, 22(10), A109 - A109.
    • Cooray S, Gilmour KC, Parsley KL, Adams S, Howe SJ, Al Ghonaium A, Bayford J, Brown L, Davies EG, Kinnon C, Gasper HB, Thrasher AJ (2011). Persistence of a polyclonal T-cell repertoire in patients with SCID-X1 following gene therapy. HUM GENE THER, 22(10), A42 - A42.
    • Mukherjee S, Thrasher AJ (2011). Progress and prospects: advancements in retroviral vector design, generation, and application.. Hum Gene Ther, 22(10), 1171 - 1174. doi:10.1089/hum.2011.2523
    • Shaw SW, David AL, Blundell M, Howe S, Pipino C, Maghsoudlou P, Lin J, Porada CD, Thrasher A, De Coppi P (2011). Sheep amniotic fluid derived CD34+stem cells engraft in NOD-SCID gamma mice and in lambs after prenatal autologous transplantation.
    • Pallant C, Cathomen T, Dannemann N, Quasim W, Naldini L, Kinnon C, Howe S, Thrasher A (2011). Targeting models of severe combined immunodeficiency for correction by zinc finger nucleases. HUM GENE THER, 22(10), A41 - A41.
    • Zhang F, Thrasher A, Antoniou M (2011). The lentiviral vector containing UCOE element driving IL2 receptor gene confers a long term immuno-reconstitution in mouse model of SCID-X1. HUM GENE THER, 22(10), A110 - A110.
    • Acosta-Saltos C, Gonitel R, Chavda S, Acosta-Saltos A, Rahim A, Thrasher A, Anderson P, Raivich G (2011). Viral expression of GM-CSF: neuroinflammatory response and effects on regeneration. HUM GENE THER, 22(10), A95 - A95.
    • Rocca C, Abdul-Razak HH, Estevez FJM, Guenechea G, Howe SJ, Holmes MC, Gregory PD, Thrasher AJ, Bueren JA, Yanez-Munoz RJ (2011). ZFN-driven gene targeting to correct mouse Prkdc scid deficiency. HUM GENE THER, 22(10), A44 - A44.
    • Bainbridge J, Sundaram V, Robbie S, Smith A, Stockman A, Rubin G, Holder G, Thrasher A, Moore A, Ali R (2011). Gene therapy for eye disease.
    • Gaspar HB, Cooray S, Gilmour KC, Parsley KL, Zhang F, Adams S, Bjorkegren E, Bayford J, Brown L, Davies EG, Veys P, Fairbanks L, Bordon V, Petropoulou T, Kinnon C, Thrasher AJ (2011). Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction.. Sci Transl Med, 3(97), 97ra80 - . doi:10.1126/scitranslmed.3002716
    • Gaspar HB, Cooray S, Gilmour KC, Parsley KL, Adams S, Howe SJ, Al Ghonaium A, Bayford J, Brown L, Davies EG, Kinnon C, Thrasher AJ (2011). Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency.. Sci Transl Med, 3(97), 97ra79 - . doi:10.1126/scitranslmed.3002715
    • Mendoza-Naranjo A, Bouma G, Pereda C, Ramírez M, Webb KF, Tittarelli A, López MN, Kalergis AM, Thrasher AJ, Becker DL, Salazar-Onfray F (2011). Functional gap junctions accumulate at the immunological synapse and contribute to T cell activation.. J Immunol, 187(6), 3121 - 3132. doi:10.4049/jimmunol.1100378
    • Mukherjee S, Thrasher AJ (2011). iPSCs: Unstable origins?. Mol Ther, 19(7), 1188 - 1190. doi:10.1038/mt.2011.116
    • Metelo J, Ward N, Thrasher AJ, Burns SO (2011). Lentivectors are efficient tools to manipulate the dendritic cell cytoskeleton.. Cytoskeleton (Hoboken), 68(8), 434 - 445. doi:10.1002/cm.20521
    • Abeyewickreme A, Thrasher AJ, Kinnon C (2011). Bone morphogenetic protein-4 (BMP4) up regulates key haematopoietic genes in differentiating embryonic stem cells treated with BMP4 short hairpin RNA.. Br J Haematol, 155(5), 638 - 641. doi:10.1111/j.1365-2141.2011.08759.x
    • Huston MW, van Til NP, Visser TP, Arshad S, Brugman MH, Cattoglio C, Nowrouzi A, Li Y, Schambach A, Schmidt M, Baum C, von Kalle C, Mavilio F, Zhang F, Blundell MP, Thrasher AJ, Verstegen MM, Wagemaker G (2011). Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning.. Mol Ther, 19(10), 1867 - 1877. doi:10.1038/mt.2011.127
    • Tolar J, Adair JE, Antoniou M, Bartholomae CC, Becker PS, Blazar BR, Bueren J, Carroll T, Cavazzana-Calvo M, Clapp DW, Dalgleish R, Galy A, Gaspar HB, Hanenberg H, Von Kalle C, Kiem HP, Lindeman D, Naldini L, Navarro S, Renella R, Rio P, Sevilla J, Schmidt M, Verhoeyen E, Wagner JE, Williams DA, Thrasher AJ (2011). Stem Cell Gene Therapy for Fanconi Anemia: Report from the 1st International Fanconi Anemia Gene Therapy Working Group Meeting. MOL THER, 19(7), 1193 - 1198. doi:10.1038/mt.2011.78
    • Bouma G, Mendoza-Naranjo A, Blundell MP, de Falco E, Parsley KL, Burns SO, Thrasher AJ (2011). Cytoskeletal remodeling mediated by WASp in dendritic cells is necessary for normal immune synapse formation and T-cell priming.. Blood, 118(9), 2492 - 2501. doi:10.1182/blood-2011-03-340265
    • Moratto D, Giliani S, Bonfim C, Mazzolari E, Fischer A, Ochs HD, Cant AJ, Thrasher AJ, Cowan MJ, Albert MH, Small T, Pai SY, Haddad E, Lisa A, Hambleton S, Slatter M, Cavazzana-Calvo M, Mahlaoui N, Picard C, Torgerson TR, Burroughs L, Koliski A, Neto JZ, Porta F, Qasim W, Veys P, Kavanau K, Hönig M, Schulz A, Friedrich W, Notarangelo LD (2011). Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study.. Blood, 118(6), 1675 - 1684. doi:10.1182/blood-2010-11-319376
    • Mandegar MA, Moralli D, Khoja S, Cowley S, Chan DY, Yusuf M, Mukherjee S, Blundell MP, Volpi EV, Thrasher AJ, James W, Monaco ZL (2011). Functional human artificial chromosomes are generated and stably maintained in human embryonic stem cells.. Hum Mol Genet, 20(15), 2905 - 2913. doi:10.1093/hmg/ddr144
    • Langford-Smith A, Wilkinson F, Langford-Smith KJ, Sergijenko A, Bennett W, Howe S, Thrasher A, Jones S, Wraith JE, Wynn R, Bigger B (2011). Lentiviral vector enhanced haematopoietic stem cell gene therapy for mucopolysaccharidosis type IIIA.
    • Bacchelli C, Buckland KF, Buckridge S, Salzer U, Schneider P, Thrasher AJ, Gaspar HB (2011). The C76R transmembrane activator and calcium modulator cyclophilin ligand interactor mutation disrupts antibody production and B-cell homeostasis in heterozygous and homozygous mice.. J Allergy Clin Immunol, 127(5), 1253 - 9.e13. doi:10.1016/j.jaci.2011.02.037
    • Thrasher AJ, Segal AW (2011). A phagocyte dilemma.... Nat Immunol, 12(3), 201 - 202. doi:10.1038/ni0311-201
    • Farmer S, Lorain S, Thrasher A, Garcia L, Muntoni F, Conti F (2011). Correction of FKRP function via RNA trans-splicing.
    • Bartholomae CC, Arens A, Balaggan KS, Yáñez-Muñoz RJ, Montini E, Howe SJ, Paruzynski A, Korn B, Appelt JU, Macneil A, Cesana D, Abel U, Glimm H, Naldini L, Ali RR, Thrasher AJ, von Kalle C, Schmidt M (2011). Lentiviral vector integration profiles differ in rodent postmitotic tissues.. Mol Ther, 19(4), 703 - 710. doi:10.1038/mt.2011.19
    • Almarza E, Zhang F, Santilli G, Blundell MP, Howe SJ, Thornhill SI, Bueren JA, Thrasher AJ (2011). Correction of SCID-X1 using an enhancerless Vav promoter.. Hum Gene Ther, 22(3), 263 - 270. doi:10.1089/hum.2010.119
    • Di WL, Larcher F, Semenova E, Talbot GE, Harper JI, Del Rio M, Thrasher AJ, Qasim W (2011). Ex-vivo Gene Therapy Restores LEKTI Activity and Corrects the Architecture of Netherton Syndrome-derived Skin Grafts. MOL THER, 19(2), 408 - 416. doi:10.1038/mt.2010.201
    • Monypenny J, Chou HC, Banon-Rodriguez I, Thrasher AJ, Anton IM, Jones GE, Calle Y (2011). Role of WASP in cell polarity and podosome dynamics of myeloid cells. EUR J CELL BIOL, 90(2-3), 198 - 204. doi:10.1016/j.ejcb.2010.05.009
    • Grez M, Reichenbach J, Schwable J, Seger R, Dinauer MC, Thrasher AJ (2011). Gene Therapy of Chronic Granulomatous Disease: The Engraftment Dilemma. MOL THER, 19(1), 28 - 35. doi:10.1038/mt.2010.232
    • Mukherjee S, Santilli G, Blundell MP, Navarro S, Bueren JA, Thrasher AJ (2011). Generation of functional neutrophils from a mouse model of X-linked chronic granulomatous disorder using induced pluripotent stem cells.. PLoS One, 6(3), e17565 - . doi:10.1371/journal.pone.0017565
    • Gaspar HB, Cooray S, Gilmour KC, Parsley KL, Zhang F, Bjorkegren E, Bayford J, Brown L, Davies EG, Kinnon C, Thrasher AJ, Adams S, Veys P, Fairbanks L, Bordon V, Petropolou T (2011). Immunodeficiency: Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction. Science Translational Medicine, 3(97), - . doi:10.1126/scitranslmed.3002716
    • Gaspar HB, Cooray S, Gilmour KC, Parsley KL, Howe SJ, Bayford J, Brown L, Davies EG, Kinnon C, Thrasher AJ, Adams S, Al Ghonaium A (2011). Immunodeficiency: Long-term persistence of a polyclonal t cell repertoire after gene therapy for X-linked severe combined immunodeficiency. Science Translational Medicine, 3(97), - . doi:10.1126/scitranslmed.3002715
    • Williams DA, Thrasher AJ (2011). Out of harm's way. Nature Biotechnology, 29(1), 41 - 42. doi:10.1038/nbt.1750
    • van der Loo JCM, Swaney WP, Grassman E, Terwilliger A, Higashimoto T, Schambach A, Baum C, Thrasher AJ, Williams DA, Nordling DL, Reeves L, Malik P (2011). Scale-up and manufacturing of clinical-grade self-inactivating γ-retroviral vectors by transient transfection. Gene Therapy, , - .
    • Ward NJ, Buckley SM, Waddington SN, Vandendriessche T, Chuah MK, Nathwani AC, McIntosh J, Tuddenham EG, Kinnon C, Thrasher AJ, McVey JH (2011). Codon optimization of human factor VIII cDNAs leads to high-level expression.. Blood, 117(3), 798 - 807. doi:10.1182/blood-2010-05-282707
    • Santilli G, Almarza E, Brendel C, Choi U, Beilin C, Blundell MP, Haria S, Parsley KL, Kinnon C, Malech HL, Bueren JA, Grez M, Thrasher AJ (2011). Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells.. Mol Ther, 19(1), 122 - 132. doi:10.1038/mt.2010.226
    • Di WL, Larcher F, Semenova E, Talbot GE, Harper JI, Del Rio M, Thrasher AJ, Qasim W (2011). Ex-vivo gene therapy restores LEKTI activity and corrects the architecture of Netherton syndrome-derived skin grafts.. Mol Ther, 19(2), 408 - 416. doi:10.1038/mt.2010.201
    • Nathwani A, Binny CJ, McIntosh JH, Tuddenham EGD, Peruta MD, Kymalainen H, Buckley S, Waddington SN, McVey JH, Spence Y, Morton CL, Thrasher A, Gray JT, Castellino FJ, Tarantal AF, Davidoff AM (2011). AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous haemorrhage.. Blood, , - .

    2010

    • Buckland K, Bachellie C, Buckridge S, Salzar U, Schneider P, Thrasher A, Gaspar B (2010). The TACI C104R mutation in humans and its murine equivalent is associated with B cell dysfunction, lympho proliferation and hypoglobulinemia.
    • Binny CJ, McIntosh JH, Tuddenham E, Waddington S, Buckley S, Thrasher A, Gray JT, Nathwani A, Tarantal AF, Davidoff A (2010). Assessing the Potential of Perinatal Gene Transfer Using Congenital Factor VII Deficiency as a Model System.
    • Macpherson L, Monypenny J, Blundell MP, Cory GO, Tome-Garcia J, Thrasher A, Jones GE, Calle Y (2010). Tyrosine Phosphorylation of WASP Promotes Calpain-Mediated Podosome Disassembly In Myeloid Cells.
    • Kane NM, Nowrouzi A, Mukherjee S, Blundell MP, Greig JA, Lee WK, Houslay MD, Milligan G, Mountford JC, von Kalle C, Schmidt M, Thrasher AJ, Baker AH (2010). Lentivirus-mediated reprogramming of somatic cells in the absence of transgenic transcription factors.. Mol Ther, 18(12), 2139 - 2145. doi:10.1038/mt.2010.231
    • Carmo M, Booth C, Montiel-Equihua CA, Schambach A, Baum C, Thrasher AJ, Gaspar HB (2010). Development of gene therapy for haemophagocytic lymphohistiocytosis (HLH) due to perforin deficiency.
    • van Til NP, Sarwari R, Visser TP, de Boer H, Li Y, Hauer J, Lagresle-Peyrou C, Danos O, Jollet A, Zhang F, Thrasher A, Cassani B, Verstegen M, Villa A, Cavazzana-Calvo M, Wagemaker G (2010). Lentiviral gene therapy of RAG1 SCID: risks and implications for treatment.
    • Qasim W, Chan E, Schaller T, Thrasher A, Towers G (2010). Lentiviral mediated restriction of HIV by TRIM-Cyclophilin fusion proteins.
    • Chick H, McDonald RA, Kritz AB, Kane NM, Alba R, Sessa WC, Thrasher AJ, Baker AH (2010). Effect of Nogo-B Overexpression in Vascular Smooth Muscle Cell Migration and Proliferation Mediated by Integration-Deficient Lentiviral Vectors.
    • Bouma G, Ancliff PJ, Thrasher AJ, Burns SO (2010). Recent advances in the understanding of genetic defects of neutrophil number and function.. Br J Haematol, 151(4), 312 - 326. doi:10.1111/j.1365-2141.2010.08361.x
    • Zhang F, Frost AR, Blundell MP, Bales O, Antoniou MN, Thrasher AJ (2010). A ubiquitous chromatin opening element (UCOE) confers resistance to DNA methylation-mediated silencing of lentiviral vectors.. Mol Ther, 18(9), 1640 - 1649. doi:10.1038/mt.2010.132
    • Westerberg LS, Meelu P, Baptista M, Eston MA, Adamovich DA, Cotta-de-Almeida V, Seed B, Rosen MK, Vandenberghe P, Thrasher AJ, Klein C, Alt FW, Snapper SB (2010). Activating WASP mutations associated with X-linked neutropenia result in enhanced actin polymerization, altered cytoskeletal responses, and genomic instability in lymphocytes. J EXP MED, 207(6), 1145 - 1152. doi:10.1084/jem.20091245
    • Qasim W, Vink CA, Thrasher AJ (2010). Hybrid lentiviral vectors.. Mol Ther, 18(7), 1263 - 1267. doi:10.1038/mt.2010.76
    • Zhang F, Antoniou MN, Thrasher A (2010). A2UCOE Lentiviral Vector Resists DNA Methylation-Mediated Silencing, Conferring Long-Term Expression in HSCs Following Serial Transplantation In Vivo.
    • Chick HE, McDonald RA, Kritz AB, Kane NM, Alba R, Sessa WC, Thrasher AJ, Baker AH (2010). Analysing the Effect of Nogo-B Overexpression in Vascular Smooth Muscle Cell Proliferation and Migration Induced by Non-Integrating or Integrating Lentiviral Vectors.
    • Ward NJ, Buckley SM, Waddington SN, Nathwani AC, Tuddenham EG, Kinnon C, Thrasher AJ, McVey JH (2010). Correction of Murine Hemophilia A Using SIN Lentiviral Vectors Expressing Enhanced Human Factor VIII cDNAs.
    • Carmo M, Booth C, Montiel-Equihua CA, Schambach A, Baum C, Thrasher AJ, Gaspar HB (2010). Development of Gene Therapy for HLH Due to Perforin Deficiency.
    • Ferreira S, Kao V, Waddington S, Fang Z, Ward N, Thrasher AJ, Antoniou MN (2010). Development of In Utero Lentiviral Vector Gene Therapy Approaches for Inherited Diseases.
    • Nibali L, O'Dea M, Bouma G, Parkar M, Thrasher AJ, Burns S, Donos N (2010). Genetic variants associated with neutrophil function in aggressive periodontitis and healthy controls.. J Periodontol, 81(4), 527 - 534. doi:10.1902/jop.2010.090543
    • Knight SB, Bokhoven M, Gubta A, Zhang F, Antoniou M, Thrasher A, Collins M, Takeuchi Y (2010). Insertional Mutagenesis by Lentiviral Vectors.
    • Chan E, Schaller T, Klein N, Thrasher A, Towers G, Qasim W (2010). Lentiviral Delivery of TRIM-Cyclophilin Restriction Factors Against HIV-1.
    • Semenova E, Larcher F, Talbot G, Harper JI, Thrasher AJ, Di WI, Qasim W (2010). Lentiviral Mediated LEKTI Expression Reverses the Netherton Syndrome Phenotype.
    • Langford-Smith AWW, Wilkinson FL, Langford-Smith KJ, Bennett W, Howe S, Thrasher A, Hemsley K, Hopwood J, Wraith JE, Wynn RF, Bigger BW (2010). Lentiviral Vector-Mediated Stem Cell Gene Therapy for Mucopolysaccharidosis Type IIIA (MPSIIIA).
    • Gonitel R, Acosta-Saltos C, T MJ, Anderson P, Raivich G, Thrasher A (2010). Non-Integrating Polycistronic Lentiviral Vectors for Use in the Central Nervous System.
    • Walshe D, Gaspar HB, Thrasher AJ, Cale CM, Gilmour KC (2010). STAT5 tyrosine phosphorylation for diagnosis and monitoring of patients with severe combined immunodeficiency.
    • Abdul-Razak HH, Rocca C, Howe SJ, Estevez FJM, Gan CHV, Roberts A, Broadstock M, Gregory PD, Holmes MC, Kinnon C, Thrasher AJ, Yanez-Munoz RJ (2010). Towards Correction of the scid Mouse by Gene Targeting.
    • Semenova E, Qasim W, Larcher F, Talbot G, Thrasher AJ, Harper JI, Di WL (2010). Translational development of ex-vivo gene therapy for Netherton syndrome.
    • Davies EG, Thrasher AJ (2010). Update on the hyper immunoglobulin M syndromes.. Br J Haematol, 149(2), 167 - 180. doi:10.1111/j.1365-2141.2010.08077.x
    • Burns SO, Killock DJ, Moulding DA, Metelo J, Nunes J, Taylor RR, Forge A, Thrasher AJ, Ivetic A (2010). A congenital activating mutant of WASp causes altered plasma membrane topography and adhesion under flow in lymphocytes.. Blood, 115(26), 5355 - 5365. doi:10.1182/blood-2009-08-236174
    • Stein S, Ott MG, Schultze-Strasser S, Jauch A, Burwinkel B, Kinner A, Schmidt M, Kramer A, Schwable J, Glimm H, Koehl U, Preiss C, Ball C, Martin H, Gohring G, Schwarzwaelder K, Hofmann WK, Karakaya K, Tchatchou S, Yang RX, Reinecke P, Kuhlcke K, Schlegelberger B, Thrasher AJ, Hoelzer D, Seger R, von Kalle C, Grez M (2010). Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. NAT MED, 16(2), 198 - U105. doi:10.1038/nm.2088
    • Loew R, Meyer Y, Kuehlcke K, Gama-Norton L, Wirth D, Hauser H, Stein S, Grez M, Thornhill S, Thrasher A, Baum C, Schambach A (2010). A new PG13-based packaging cell line for stable production of clinical-grade self-inactivating γ-retroviral vectors using targeted integration. Gene Therapy, 17(2), 272 - 280. doi:10.1038/gt.2009.134
    • Picard C, Von Bernuth H, Ghandil P, Chrabieh M, Ku C-L, Abel L, Puel A, Casanova J-L, Al-Muhsen S, Al-Hajjar S, Al-Ghonaium A, Levy O, McDonald D, Geha RS, Arkwright PD, Takada H, Hara T, Krause JC, Creech CB, Ehl S, Maródi L, Day-Good NK, Holland SM, Gallin JI, Chapel H, Speert DP, Rodriguez-Gallego C, Colino E, Garty B-Z, Roifman C, Yoshikawa H, Nonoyama S, Domachowske J, Issekutz AC, Tang M, Smart J, Zitnik SE, Hoarau C, Kumararatne DS, Doffinger R, Thrasher AJ, Davies EG, Bethune C, Sirvent N, De Ricaud D, Camcioglu Y, Vasconcelos J, Guedes M, Vitor AB, Rodrigo C, Almazán F, Méndez M, Aróstegui JI, Alsina L, Fortuny C, Reichenbach J, Verbsky JW, Bossuyt X (2010). Clinical features and outcome of patients with IRAK-4 and MyD88 deficiency. Medicine, 89(6), 403 - 425. doi:10.1097/MD.0b013e3181fd8ec3
    • Montiel-Equihua CA, Thrasher AJ, Gaspar HB (2010). Development of gene therapy: Potential in severe combined immunodeficiency due to adenosine deaminase deficiency. Stem Cells and Cloning: Advances and Applications, 3(1), 1 - 12.
    • Georgiadis A, Tschernutter M, Bainbridge JW, Balaggan KS, Mowat F, West EL, Munro PM, Thrasher AJ, Matter K, Balda MS, Ali RR (2010). The tight junction associated signalling proteins ZO-1 and ZONAB regulate retinal pigment epithelium homeostasis in mice.. PLoS One, 5(12), e15730 - . doi:10.1371/journal.pone.0015730
    • Blundell MP, Worth A, Bouma G, Thrasher AJ (2010). The Wiskott-Aldrich syndrome: The actin cytoskeleton and immune cell function.. Dis Markers, 29(3-4), 157 - 175. doi:10.3233/DMA-2010-0735
    • Williams DA, Thrasher AJ, Baum C (2010). Transatlantic consortium spotlights need for changes in gene therapy trials. Molecular Therapy, 18(11), 1892 - 1892. doi:10.1038/mt.2010.227
    • Albert MH, Bittner TC, Nonoyama S, Notarangelo LD, Burns S, Imai K, Espanol T, Fasth A, Pellier I, Strauss G, Morio T, Gathmann B, Noordzij JG, Fillat C, Hoenig M, Nathrath M, Meindl A, Pagel P, Wintergerst U, Fischer A, Thrasher AJ, Belohradsky BH, Ochs HD (2010). X-linked thrombocytopenia (XLT) due to WAS mutations: clinical characteristics, long-term outcome, and treatment options. BLOOD, 115(16), 3231 - 3238. doi:10.1182/blood-2009-09-239087
    • Mukherjee S, Santilli G, Blundell MP, Almarza E, Baker A, Bueren J, Thrasher AJ (2010). Gene-Corrected Neutrophils from Induced Pluripotent Stem Cells Derived from an X-CGD Disease Model.
    • Kane NM, Nowrouzi A, Greig JA, Houslay MD, Milligan G, Mountford JC, Von Kalle C, Thrasher AJ, Schmidt M, Baker AH (2010). Induction of iPS cells from Dermal Fibroblasts by High Dose HIV-Based LV Vectors in the Absence of Reprogramming Factors: Analysis of Integration Sites and Genomic Stability.
    • Thrasher AJ, Burns SO (2010). WASP: a key immunological multitasker.. Nat Rev Immunol, 10(3), 182 - 192. doi:10.1038/nri2724
    • Ginn SL, Liao SH, Dane AP, Zheng M, Alexander IE, Hu M, Alexander SI, Hyman J, Finnie JW, Cavazzana-Calvo M, Thrasher AJ (2010). Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and γc overexpression. Molecular Therapy, 18(5), 965 - 976. doi:10.1038/mt.2010.50

    2009

    • Blundell MP, Bouma G, Metelo J, Worth A, Calle Y, Cowell LA, Westerberg LS, Moulding DA, Mirando S, Kinnon C, Cory GO, Jones GE, Snapper SB, Burns SO, Thrasher AJ (2009). Phosphorylation of WASp is a key regulator of activity and. Proc Natl Acad Sci U S A, 106(37), 15738 - 15743.
    • Bouma G, Burns SO, Thrasher AJ (2009). Wiskott-Aldrich Syndrome: Immunodeficiency resulting from defective cell migration and impaired immunostimulatory activation. Immunobiology, 214(9-10), 778 - 790.
    • Wu Y, Wu W, Wong WM, Ward E, Thrasher AJ, Goldblatt D, Osman M, Digard P, Canaday DH, Gustafsson K (2009). Human gamma delta T cells: a lymphoid lineage cell capable of. J Immunol, 183(9), 5622 - 5629.
    • Wu Y, Wu W, Wong WM, Ward E, Thrasher AJ, Goldblatt D, Osman M, Digard P, Canaday DH, Gustafsson K (2009). Human gammadelta T cells: a lymphoid lineage cell capable of professional phagocytosis. The Journal of Immunology, 183(9), 5622 - 5629.
    • Qasim W, Gaspar HB, Thrasher AJ (2009). Progress and prospects: gene therapy for inherited immunodeficiencies. Gene Ther., , - .
    • Blundell MP, Bouma G, Metelo J, Worth A, Calle Y, Cowell LA, Westerberg LS, Moulding DA, Mirando S, Kinnon C, Cory GO, Jones GE, Snapper SB, Burns SO, Thrasher AJ (2009). Phosphorylation of WASp is a key regulator of activity and stability in vivo. Proc.Natl.Acad.Sci.U.S.A, 106(37), 15738 - 15743.
    • Thrasher A (2009). Regenerative Medicine for Primary Immunodeficiencies. HUMAN GENE THERAPY, 20(9), 1031 - 1031.
    • Modlich U, Navarro S, Zychlinski D, Maetzig T, Knoess S, Brugman MH, Schambach A, Charrier S, Galy A, Thrasher AJ, Bueren J, Baum C (2009). Insertional Transformation of Hematopoietic Cells by Self-inactivating Lentiviral and Gammaretroviral Vectors. Mol.Ther., , - .
    • Bouma G, Doffinger R, Patel SY, Peskett E, Sinclair JC, Barcenas-Morales G, Cerron-Gutierrez L, Kumararatne DS, Graham DE, Thrasher AJ, Burns SO (2009). Impaired neutrophil migration and phagocytosis in IRAK-4 deficiency. Br.J.Haematol., , - .
    • Adamovich DA, Nakamura F, Worth A, Burns S, Thrasher AJ, Hartwig JH, Snapper SB (2009). Activating mutations of N-WASP alter Shigella pathogenesis. Biochem.Biophys.Res.Commun., 384(3), 284 - 289.
    • Scobie L, Hector RD, Grant L, Bell M, Nielsen AA, Meikle S, Philbey A, Thrasher AJ, Cameron ER, Blyth K, Neil JC (2009). A novel model of SCID-X1 reconstitution reveals predisposition to retrovirus-induced lymphoma but no evidence of gammaC gene oncogenicity. Mol.Ther., 17(6), 1031 - 1038.
    • Cooper N, Davies EG, Thrasher AJ (2009). Repeated courses of rituximab for autoimmune cytopenias may precipitate profound hypogammaglobulinaemia requiring replacement intravenous immunoglobulin. Br.J.Haematol., 146(1), 120 - 122.
    • Mukherjee S, Blundell MP, Bouma G, Kinnon C, Thrasher AJ (2009). A Comparative Study of Non-Integrating and Integrating Lentiviral Vectors in Eliciting an Effective Immune Response Against Hepatitis B Surface Antigen in Mice.
    • Rivat C, Booth C, Osman M, Thrasher A, Gaspar B (2009). Development of Gene Therapy for X-Linked Lymphoproliferative Disease.
    • Qasim W, Talbot G, Larcher F, Harper J, Thrasher A, Di WL (2009). Development of Lentiviral Therapy for Netherton Syndrome.
    • Rahim AA, Wong AM, Buckley SM, Hughes DA, Karlsson S, Cooper JD, Thrasher AJ, Mehta A, Waddington SN (2009). Fetal Gene Therapy for Acute Neuronopathic Gaucher Disease.
    • Paruzynski A, Gabriel R, Eckenberg R, Bartholomae CC, Nowrouzi A, Arens A, Howe SJ, Recchia A, Cattoglio C, Wang W, Schwarzwaelder K, Kirsten R, Deichmann A, Ball CR, Balaggan KS, Yanez-Munoz RJ, Ali RR, Gaspar HB, Biasco L, Aiuti A, Cesana D, Montini E, Naldini L, Cohen-Haguenauer O, Mavilio F, Thrasher AJ, Glimm H, von Kalle C, Saurin W, Schmidt M (2009). Modeling and Increasing the Genome Accessibility of Viral Integration Sites.
    • Ward NJ, Apolonia L, Cochrane M, Buckley SM, Waddington SN, Nathwani AC, Philpott NJ, Kinnon C, Thrasher AJ (2009). Nonintegrating Lentiviral Vectors for Treatment of Haemophilia B.
    • Karwacz K, Apolonia L, Escors D, Thrasher A, Collins M (2009). Non-Integrating Lentiviral Vectors for Vaccination.
    • Zhang F, Antoniou MN, Thrasher AJ (2009). Ubiquitous Chromatin Opening Element (UCOE)-Driven Transgenes within Lentiviral Vectors Are Not Subject to DNA Methylation Mediated Silencing.
    • Zanta-Boussif MA, Charrier S, Brice-Ouzet A, Martin S, Opolon P, Thrasher AJ, Hope TJ, Galy A (2009). Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS. Gene Ther., 16(5), 605 - 619.
    • Giannakopoulos A, Stavrou EF, Zarkadis I, Zoumbos N, Thrasher AJ, Athanassiadou A (2009). The functional role of S/MARs in episomal vectors as defined by the stress-induced destabilization profile of the vector sequences. J.Mol.Biol., 387(5), 1239 - 1249.
    • Locci M, Draghici E, Marangoni F, Bosticardo M, Catucci M, Aiuti A, Cancrini C, Marodi L, Espanol T, Bredius RG, Thrasher AJ, Schulz A, Litzman J, Roncarolo MG, Casorati G, Dellabona P, Villa A (2009). The Wiskott-Aldrich syndrome protein is required for iNKT cell maturation and function. J.Exp.Med., 206(4), 735 - 742.
    • Rahim AA, Wong AM, Howe SJ, Buckley SM, costa-Saltos AD, Elston KE, Ward NJ, Philpott NJ, Cooper JD, Anderson PN, Waddington SN, Thrasher AJ, Raivich G (2009). Efficient gene delivery to the adult and fetal CNS using pseudotyped non-integrating lentiviral vectors. Gene Ther., 16(4), 509 - 520.
    • Karwacz K, Mukherjee S, Apolonia L, Blundell MP, Bouma G, Escors D, Collins MK, Thrasher AJ (2009). Nonintegrating lentivector vaccines stimulate prolonged T-cell and antibody responses and are effective in tumor therapy. J.Virol., 83(7), 3094 - 3103.
    • Karwacz K, Mukherjee S, Apolonia L, Blundell MP, Bouma G, Escors D, Collins MK, Thrasher AJ (2009). Nonintegrating lentivector vaccines stimulate prolonged T-cell and antibody responses and are effective in tumor therapy.. J Virol, 83(7), 3094 - 3103. doi:10.1128/JVI.02519-08
    • Gjertsson I, Laurie KL, Devitt J, Howe SJ, Thrasher AJ, Holmdahl R, Gustafsson K (2009). Tolerance induction using lentiviral gene delivery delays onset and severity of collagen II arthritis. Mol.Ther., 17(4), 632 - 640.
    • Walshe D, Gaspar HB, Thrasher AJ, Cale CM, Gilmour KC (2009). Signal transducer and activator of transcription 5 tyrosine phosphorylation for the diagnosis and monitoring of patients with severe combined immunodeficiency. J.Allergy Clin.Immunol., 123(2), 505 - 508.
    • Karwacz K, Mukherjee S, Apolonia L, Blundell MP, Bouma G, Escors D, Collins MK, Thrasher AJ (2009). Non-integrating Lentivector Vaccines Stimulate Prolonged T Cell and Antibody Responses and are Effective in Tumor Therapy. J.Virol., , - .
    • Modlich U, Knoss S, Navarro S, Zychlinski D, Matzig T, Brugman M, Schambach A, Thrasher A, Bueren J, Grez M, Baum C (2009). Assessing genotoxicity of lenti- and gammaretroviral vectors in vitro. Human Gene Therapy, 20(11), 1508 - 1508.
    • Gabriel R, Eckenberg R, Paruzynski A, Bartholomae CC, Nowrouzi A, Arens A, Howe SJ, Recchia A, Cattoglio C, Wang W, Faber K, Schwarzwaelder K, Kirsten R, Deichmann A, Ball CR, Balaggan KS, Yanez-Munoz RJ, Ali RR, Gaspar HB, Biasco L, Aiuti A, Cesana D, Montini E, Naldini L, Cohen-Haguenauer O, Mavilio F, Thrasher AJ, Glimm H, von Kalle C, Saurin W, Schmidt M (2009). Comprehensive genomic access to vector integration in clinical gene therapy. Nature Medicine, 15(12), 1431 - 1U13.
    • Antoniou M, Montiel-Equihua C, Monkeviciute A, Thrasher A, Gaspar B (2009). Development of a lentiviral vector for systemic gene therapy of SCID-ADA. Human Gene Therapy, 20(11), 1467 - 1468.
    • Stein S, Ott M, Schultze-Strasser S, Kinner A, Jauch A, Burwinkel B, Schmidt M, Kramer A, Martin H, Glimm H, Koehl U, Preiss C, Rudolf E, Kunkel H, Gohring G, Schwarzwaelder K, Hofmann WK, Kuhlcke K, Schlegelberger B, Thrasher A, Hoelzer D, Seger R, von Kalle C, Grez M (2009). Gene therapy for chronic granulomatous disease: Ups and downs. Human Gene Therapy, 20(11), 1440 - 1441.
    • Bouma G, Peskett E, Sinclair JC, Graham Davies E, Thrasher AJ, Burns SO, Doffinger R, Patel SY, Cerron-Gutierrez L, Kumararatne DS, Barcenas-Morales G (2009). Impaired neutrophil migration and phagocytosis in IRAK-4 deficiency: Correspondence. British Journal of Haematology, 147(1), 153 - 156. doi:10.1111/j.1365-2141.2009.07838.x
    • Howe SJ, Mansour MR, Schwarzwaelder K, Bartholomae C, Hubank M, Kempski H, Brugman MH, Pike O, K C, S D, Ridder D, Gilmour KC, Adams S, Thornhill SI, Parsley KL, Staal FJ, Gale RE, Linch DC, Bayford J, Brown L, Quaye M, Kinnon C, Ancliff P, Webb DK, Schmidt M, von KC, Gaspar HB, Thrasher AJ (2009). Insertional mutagenesis combined with acquired somatic mutations causes leukaemogenesis following gene therapy of SCID-X1 patients. Journal of Clinical Investigation, 118(9), 3143 - 3150.
    • Paruzynski A, Arens A, Gabriel R, Bartholomae C, Eckenberg R, Nowrouzi A, Scholz S, Howe S, Recchia A, Cattoglio C, Wang W, Faber K, Schwarzwaelder K, Kirsten R, Deichmann A, Ball C, Balaggan K, Yanez-Munoz R, Ali R, Gaspar HB, Biasco L, Aiuti A, Cesana D, Montini E, Naldini L, Cohen-Haguenauer O, Mavilio F, Thrasher A, Glimm H, von Kalle C, Saurin W, Schmidt M (2009). Integration site analysis by non-restrictive (nr) LAM-PCR combined with optimized high-throughput sequencing. Human Gene Therapy, 20(11), 1509 - 1510.
    • Ginn S, Liao S, Dane A, Smyth C, Hyman J, Finnie J, Zheng ML, Thrasher A, Alexander I (2009). Lymphomagenesis in SCID-X1 mice independent of insertional mutagenesis and gamma c over-expression. Human Gene Therapy, 20(11), 1462 - 1462.
    • Thrasher AJ (2009). New insights into the biology of Wiskott-Aldrich syndrome. Hematology Am Soc Hematol.Educ Program., , 132 - 138.
    • Mukherjee S, Apolonia L, Blundell MP, Bouma G, Thrasher AJ, Karwacz K, Escors D, Collins MK (2009). Nonintegrating lentivector vaccines stimulate prolonged T-cell and antibody responses and are effective in tumor therapy. Journal of Virology, 83(7), 3094 - 3103. doi:10.1128/JVI.02519-08
    • Howe S, Bartolovic K, Milsom M, Thrasher A, Kinnon C (2009). Optimisation of integration-deficient lentivirus (IDLV) application in hematopoietic stem cells. Human Gene Therapy, 20(11), 1463 - 1463.
    • Vink CA, Gaspar HB, Gabriel R, Schmidt M, McIvor RS, Thrasher AJ, Qasim W (2009). Sleeping Beauty Transposition From Nonintegrating Lentivirus. Molecular Therapy, 17(7), 1197 - 1204.
    • Moreno-Carranza B, Gentsch M, Stein S, Schambach A, Santilli G, Rudolf E, Ryser MF, Haria S, Thrasher AJ, Baum C, Brenner S, Grez M (2009). Transgene optimization significantly improves SIN vector titers, gp91phox expression and reconstitution of superoxide production in X-CGD cells. Gene Ther., 16(1), 111 - 118.
    • Bouma G, Burns SO, Thrasher AJ (2009). Wiskott-Aldrich Syndrome: Immunodeficiency resulting from. Immunobiology, 214(9-10), 778 - 790.
    • Stein S, Ott M, Schultze-Strasser S, Kinner A, Jauch A, Burwinkel B, Schmidt M, Kramer A, Martin H, Glimm H, Koehl U, Preiss C, Rudolf E, Kunkel H, Gohring G, Schwarzwaelder K, Hofmann WK, Kuhlcke K, Schlegelberger B, Thrasher A, Hoelzer D, Seger R, von Kalle C, Grez M (2009). Gene therapy for chronic granulomatous disease: Ups and downs.

    2008

    • Bowna G, Doffinger R, Patel S, Peskett E, Sinclair J, Kumararatne D, Davies EG, Thrasher AJ, Burns S (2008). Neutrophil dysfunction 2 in IRAK-(4) deficiency.
    • Calle Y, Anton IM, Thrasher AJ, Jones GE (2008). WASP and WIP regulate podosomes in migrating leukocytes. Journal of Microscopy, 231(3), 494 - 505.
    • Burns S, Hardy S, Bouma G, Klein N, Thrasher AJ (2008). Dendritic cells require CD18 for normal podosome formation.
    • Stein S, Ott MG, Schultze-Strasser S, Jauch A, Burwinkel B, Schmidt M, Martin H, Koehl U, Preiss C, Rudolf E, Kunkel H, Schwarzwaelder K, Hofmann WK, Kuehlcke K, Schlegelberger B, Thrasher AJ, Seger R, von Kalle C, Hoelzer D, Grez M (2008). Gene therapy for chronic granulomatous disease: Current status of the German clinical study.
    • Knight S, Bokhoven M, Gupta A, Ceylan M, Zhang F, Antoniou M, Thrasher A, Takeuchi Y, Collins M (2008). Insertional mutagenesis by retroviral and lentiviral vectors.
    • Kao V, Waddington S, Thrasher A, Antoniou M (2008). Novel lentiviral vectors for haemophilia gene therapy.
    • Zhang F, Antoniou M, Thrasher A (2008). Ubiquitous chromatin opening element (UCOE)-driven transgenes within lentiviral vectors are not subject to DNA methylation mediated silencing.
    • Moreno-Carranza B, Gentsch M, Stein S, Schambach A, Santilli G, Rudolf E, Ryser MF, Haria S, Thrasher AJ, Baum C, Brenner S, Grez M (2008). Transgene optimization significantly improves SIN vector titers, gp91(phox) expression and reconstitution of superoxide production in X-CGD cells. Gene Ther., , - .
    • Howe SJ, Mansour MR, Schwarzwaelder K, Bartholomae C, Hubank M, Kempski H, Brugman MH, Pike OK, Chatters S, de RD, Gilmour KC, Adams S, Thornhill SI, Parsley KL, Staal FJ, Gale RE, Linch DC, Bayford J, Brown L, Quaye M, Kinnon C, Ancliff P, Webb DK, Schmidt M, von KC, Gaspar HB, Thrasher A (2008). Insertional mutagenesis combined with acquired somatic mutations causes leukaemogenesis following gene therapy of SCID-X1 patients. Journal of Clinical Investigation, 118(9), 3143 - 3150.
    • Buckley SM, Howe SJ, Sheard V, Ward NJ, Coutelle C, Thrasher AJ, Waddington SN, McKay TR (2008). Lentiviral transduction of the murine lung provides efficient pseudotype and developmental stage-dependent cell-specific transgene expression. Gene Ther., 15(16), 1167 - 1175.
    • Bartholomae CC, Yanez-Munoz RJ, Balaggan KS, Howe SJ, MacNeil A, Ali RR, Thrasher AJ, von Kalle C, Schmidt M (2008). Nearly random integration site distribution in postmitotic rodent tissues transduced with lentiviral SIN-vectors. Blood Cells, Molecules, and Diseases, 40(2), 6 - .
    • Titman P, Pink E, Skucek E, O'Hanlon K, Cole TJ, Gaspar J, Xu-Bayford J, Jones A, Thrasher AJ, Davies EG, Veys PA, Gaspar HB (2008). Cognitive and behavioural abnormalities in children following haematopoietic stem cell transplantation for severe congenital immunodeficiencies. Blood, , - .
    • Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K, Viswanathan A, Holder GE, Stockman A, Tyler N, Petersen-Jones S, Bhattacharya SS, Thrasher AJ, Fitzke FW, Carter BJ, Rubin GS, Moore AT, Ali RR (2008). Effect of gene therapy on visual function in Leber\'s congenital amaurosis. N.Engl.J.Med., 358(21), 2231 - 2239.
    • Thrasher AJ (2008). Gene therapy for primary immunodeficiencies.. Immunol Allergy Clin North Am, 28(2), 457 - xi. doi:10.1016/j.iac.2008.02.001
    • Blundell MP, Bouma G, Calle Y, Jones GE, Kinnon C, Thrasher AJ (2008). Improvement of migratory defects in a murine model of Wiskott-Aldrich syndrome gene therapy. MOL THER, 16(5), 836 - 844. doi:10.1038/mt.2008.43
    • Jones LB, McGrogan P, Flood TJ, Gennery AR, Morton L, Thrasher A, Goldblatt D, Parker L, Cant AJ (2008). Special article: chronic granulomatous disease in the United Kingdom and Ireland: a comprehensive national patient-based registry. Clinical and Experimental Dermatology, 152(2), 211 - 218.
    • Rahim AA, Waddington SN, Wong AMS, Ward NJ, Elston KE, Buckley SMK, Philpott NJ, Cooper JD, Anderson PN, Thrasher AJ, Raivich G (2008). Efficient gene delivery to the adult and fetal central nervous system using pseudotyped non-integrating lentiviral vectors.
    • Santilli G, Blundell M, Haria S, Kinnon C, Antoniou M, Grez M, Thrasher A (2008). Evaluation of self-inactivating lentiviral vectors containing myeloid specific promoters in combination with the ubiquitously acting chromatin opening element (UCOE) for gene therapy of X-CGD.
    • Santilli G, Thornhill SI, Kinnon C, Thrasher AJ (2008). Gene therapy of inherited immunodeficiencies. Expert.Opin.Biol Ther, 8(4), 397 - 407.
    • Howe SJ, Mansour M, Brugman M, Pike-Overzet K, Hubank M, Jina N, de Ridder D, Staal F, Baum C, Kinnon C, Gaspar HB, Thrasher AJ (2008). Molecular analysis of a severe adverse event in the UKSCID-X1 gene therapy clinical trial.
    • Ward NJ, Apolonia L, Fernandes C, Waddington SN, Cochrane M, Nathwani AC, Thrasher AJ, Kinnon C, Philpott NJ (2008). Non-integrating lentiviral vector pseudotypes for safer gene therapy.
    • Kao V, Waddington S, Thrasher A, Antoniou M (2008). Novel lentiviral vectors for targeting the red blood cell lineage. HUMAN GENE THERAPY, 19(4), 415 - 415.
    • Talbot GE, Qasim W, Thrasher AJ, Harper JI, Di WL (2008). Reproducible and stable transgene expression towards ex vivo lentiviral gene therapy for Netherton syndrome.
    • Talbot GE, Qasim W, Thrasher AJ, Harper JI, Di WL (2008). Reproducible and stable transgene expression towards ex vivo lentiviral gene therapy for Netherton Syndrome.
    • Thornhill SI, Grassman E, Ulaganathan M, Waddington SN, Howe SJ, Van Der Loo H, Schambach A, Kinnon C, Baum C, Williams D, Thrasher AJ (2008). Self-inactivating retroviral vectors for gene therapy of SCID-X1.
    • Blundell MP, Bouma G, Calle Y, Jones GE, Kinnon C, Thrasher AJ (2008). Improvement of Migratory Defects in a Murine Model of Wiskott-Aldrich Syndrome Gene Therapy. Mol.Ther, , - .
    • Deichman A, Schmidt M, Abina SHB, Cavazzana-Calvo M, Schwarzwaelder K, Howe SJ, Thrasher AJ, Glimm H, Ott MG, Hoelzer D, Grez M, Seger R, Aiuti A, von Kalle CN (2008). Comparative integration site profile analysis in 5 clinical retroviral gene therapy studies. doi:10.1016/j.bcmd.2007.10.033
    • Bartholomae CC, Yanez-Munoz RJ, Balaggan KS, Howe SJ, MacNeil A, Ali RR, Thrasher AJ, von Kalle C, Schmidt M (2008). Nearly random integration site distribution in postmitotic rodent tissues transduced with lentiviral SIN-vectors. doi:10.1016/j.bcmd.2007.10.016
    • Gaspar HB, Bjorkegren E, Parsley K, Gilmour K, Fairbanks L, Zhang F, Giannakopoulos A, King D, Adams S, Bayford JX, Henderson L, Davies G, Veys P, Thrasher AJ (2008). Reconstitution of immune function in adenosine deaminase deficient severe combined immunodeficiency following hematopoietic stem cell gene therapy. doi:10.1016/j.bcmd.2007.10.041
    • Thornhill SI, Schambach A, Howe SJ, Ulaganathan M, Grassman E, Williams D, Schiedlmeier B, Sebire NJ, Gaspar HB, Kinnon C, Baum C, Thrasher AJ (2008). Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency.. Mol Ther, 16(3), 590 - 598. doi:10.1038/sj.mt.6300393
    • Toscano MG, Frecha C, Benabdellah K, Cobo M, Blundell M, Thrasher AJ, Garcia-Olivares E, Molina IJ, Martin F (2008). Hematopoietic-specific lentiviral vectors circumvent cellular toxicity due to ectopic expression of Wiskott-Aldrich syndrome protein. HUM GENE THER, 19(2), 179 - 197. doi:10.1089/hum.2007.098
    • Gabriel R, Paruzynski A, Eckenberg R, Bartholomae CC, Nowrouzi A, Wang W, Schwarzwaelder K, Arens A, Kirsten R, Deichmann A, Ball CR, Howe SJ, Recchia A, Claudia C, Balaggan KS, Yanez-Munoz RJ, Ali RR, Mavilio F, Gaspar HB, Thrasher AJ, Glimm H, von Kalle C, Saurin W, Schmidt M (2008). Assessing the genome accessibility of viral integration sites. Human Gene Therapy, 19(10), 1085 - 1085.
    • Himoudi N, Nabarro S, Buddle J, Eddaoudi A, Thrasher AJ, Anderson J (2008). Bone marrow-derived IFN-producing killer dendritic cells account for the tumoricidal activity of unpulsed dendritic cells. J.Immunol., 181(9), 6654 - 6663.
    • Titman P, Pink E, Skucek E, O'Hanlon K, Cole TJ, Gaspar J, Xu-Bayford J, Jones A, Thrasher AJ, Davies EG, Gaspar HB, Veys PA (2008). Cognitive and behavioral abnormalities in children after hematopoietic stem cell transplantation for severe congenital immunodeficiencies. Blood, 112(9), 3907 - 3913. doi:10.1182/blood-2008-04-151332
    • Deichman A, Schmidt M, Abina SHB, Cavazzana-Calvo M, Schwarzwaelder K, Howe SJ, Thrasher AJ, Glimm H, Ott MG, Hoelzer D, Grez M, Seger R, Aiuti A, von Kalle CN (2008). Comparative integration site profile analysis in 5 clinical retroviral gene therapy studies. Blood Cells, Molecules, and Diseases, 40(2), 23 - .
    • Galy A, Roncarolo MG, Thrasher AJ (2008). Development of lentiviral gene therapy for Wiskott Aldrich syndrome. Expert Opinion on Biological Therapy, 8(2), 181 - 190.
    • Thornhill SI, Thrasher AJ (2008). Gene therapy for severe combined immunodeficiency: Success and risk. Biochemist, 30(3), 26 - 29.
    • Howe SJ, Mansour M, Brugman M, Pike-Overzet K, Hubank M, Jina N, de Ridder D, Staal F, Baum C, Kinnon C, Gaspar HB, Thrasher AJ (2008). Molecular analysis of a severe adverse event in the UKSCID-X1 gene therapy clinical trial. Human Gene Therapy, 19(4), 82 - .
    • Buckley SM, Howe SJ, Rahim AA, Buning H, McIntosh J, Wong SP, Baker AH, Nathwani A, Thrasher AJ, Coutelle C, McKay TR, Waddington SN (2008). Luciferin Detection After Intranasal Vector Delivery Is Improved by Intranasal Rather Than Intraperitoneal Luciferin Administration.. Human Gene Therapy, 19(10), 1050 - 1056.
    • Worth AJJ, Cory GOC, Thrasher AJ, Burns S (2008). Molecular pathogenesis of Wiskott-Aldrich syndrome mutations: insights into the mechanisms of endritic cell migration.
    • Salzer U, Bacchelli C, Buckridge S, Pan-Hammarstrom Q, Jennings S, Lougaris V, Bergbreiter A, Hagena T, Birmelin J, Plebani A, Webster AD, Peter HH, Suez D, Chapel H, Lean-Tooke A, Spickett GP, nover-Sombke S, Ochs HD, Urschel S, Belohradsky BH, Ugrinovic S, Kumararatne DS, Lawrence TC, Holm AM, Franco JL, Schulze I, Schneider P, Gertz EM, Schaffer AA, Hammarstrom L, Thrasher AJ, Gaspar HB, Grimbacher B (2008). Relevance of biallelic versus monoallelic TNFRSF13B mutations in distinguishing disease-causing from risk-increasing TNFRSF13B variants in antibody deficiency syndromes. Blood, , - .
    • Gabriel R, Paruzynski A, Eckenberg R, Bartholomae CC, Nowrouzi A, Wang W, Schwarzwaelder K, Arens A, Kirsten R, Deichmann A, Ball C, Howe SJ, Recchia A, Cattoglio C, Balaggan KS, Yanez-Munoz RJ, Ali RR, Mavilio F, Gaspar B, Thrasher A, Glimm H, von Kalle C, Saurin W, Schmidt M (2008). Comprehensive and Unbiased Integration Site Analysis in Clinical Gene Therapy.
    • Meyer-Bahlburg A, Becker-Herman S, Humblet-Baron S, Khim S, Weber M, Bouma G, Thrasher AJ, Batista FD, Rawlings DJ (2008). Wiskott-Aldrich syndrome protein deficiency in B cells results in impaired peripheral homeostasis. Blood, 112(10), 4158 - 4169.
    • Salzer US, Bacchelli C, Buckridge S, Pan-Hammar-Strom Q, Jennings S, Lougaris V, Bergbreiter A, Hagena T, Birmelin J, Plebani A, Webster ADB, Peter HH, Suez D, Chapel H, Maclean-Tooke A, Spickett GP, Anover-Sombke S, Ochs HD, Urschel S, Belohradsky BH, Ugrinovic S, Kumararatne DS, Lawrence TC, Holm AM, Franco JL, Schulze I, Schneider P, Gertz ME, Schaeffer AA, Hammarstrom L, Thrasher AJ (2008). Relevance of biallelic vs monoallelic TNFRSF13B mutations in distinguishing disease-causing from risk-increasing TNFRSF13B variants in antibody deficiency syndromes. CLINICAL AND EXPERIMENTAL IMMUNOLOGY, 154, 207 - 208.
    • Himoudi N, Nabarro S, Buddle J, Eddaoudi A, Thrasher AJ, Anderson J (2008). Bone marrow-derived IFN-producing killer dendritic cells account for the tumoricidal activity of unpulsed dendritic cells. J Immunol, 181(9), 6654 - 6663.
    • Himoudi N, Nabarro S, Buddle J, Eddaoudi A, Thrasher AJ, Anderson J (2008). Bone marrow-derived IFN-producing killer dendritic cells account for the tumoricidal activity of unpulsed dendritic cells.. J Immunol, 181(9), 6654 - 6663.
    • Titman P, Pink E, Skucek E, O'Hanlon K, Cole TJ, Gaspar J, Xu-Bayford J, Jones A, Thrasher AJ, Davies EG, Veys PA, Gaspar HB (2008). Cognitive and behavioral abnormalities in children after hematopoietic stem cell transplantation for severe congenital immunodeficiencies. Blood, 112(9), 3907 - 3913.
    • Thornhill SI, Schambach A, Howe SJ, Ulaganathan M, Grassman E, Williams D, Schiedlmeier B, Sebire NJ, Gaspar HB, Kinnon C, Baum C, Thrasher AJ (2008). Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency. Molecular Therapy, 16(3), 590 - 598.
    • Thornhill SI, Grassman E, Ulaganathan M, Waddington SN, Howe SJ, Van Der Loo H, Schambach A, Kinnon C, Baum C, Williams D, Thrasher AJ (2008). Self-inactivating retroviral vectors for gene therapy of SCID-X1. Human Gene Therapy, 19(4), O15 - .
    • Schwarzwaelder K, Schmidt M, Deichmann A, Howe S, Ott MG, Stein S, Siler U, Hacein-Bey-Abina S, Glimm H, Hoelzer D, Cavazzana-Calvo M, Thrasher A, Seger R, Grez M, von Kalle C (2008). The gene corrected clonal inventory in clinical gene therapy trials. Blood Cells, Molecules, and Diseases, 40(2), 66 - .
    • Schwarzwaelder K, Schmidt M, Deichmann A, Howe S, Ott MG, Stein S, Siler U, Hacein-Bey-Abina S, Glimm H, Hoelzer D, Cavazzana-Calvo M, Thrasher A, Seger R, Grez M, von Kalle C (2008). The gene corrected clonal inventory in clinical gene therapy trials. doi:10.1016/j.bcmd.2007.10.076
    • Ozsahin H, Cavazzana-Calvo M, Notarangelo LD, Schulz A, Thrasher AJ, Mazzolari E, Slatter MA, Le Deist F, Blanche S, Veys P, Fasth A, Bredius R, Sedlacek P, Wulffraat N, Ortega J, Heilmann C, O'Meara A, Wachowiak J, Kalwak K, Matthes-Martin S, Ikinciogullari A, Cant AJ, Friedrich W, Fischer A (2008). Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation.. Blood, 111(1), 439 - 445.

    2007

    • Buckley SMK, Howe SJ, Thrasher A, Baker AH, Mckay TR, Coutelle C, Waddington S (2007). The route of luciferin administration is a critical determinant of detection of pulmonary and nasal luciferase expression by in vivo bioimaging.
    • Gaspar B, Thrasher A (2007). Successful bone marrow gene therapy for severe combined immunodeficiency. Inmunologia, 26(4), 218 - 219.
    • Kim JJ, Thrasher AJ, Jones AM, Davies EG, Cale CM (2007). Rituximab for the treatment of autoimmune cytopenias in children with immune deficiency. BRITISH JOURNAL OF HAEMATOLOGY, 138(1), 94 - 96. doi:10.1111/j.1365-2141.2007.06616.x
    • Bouma G, Burns SO, Thrasher AJ (2007). Impaired T cell priming in vivo resulting from dysfunction of WASp-deficient dendritic cells. Blood, 110(3), 4278 - 4284. doi:10.1182/blood-2007-06-096875
    • Alexander BL, Ali RR, Alton EWFW, Bainbridge JW, Braun S, Cheng SH, Flotte TR, Gaspar HB, Grez M, Griesenbach U, Kaplitt MG, Ott MG, Seger R, Simons M, Thrasher AJ, Thrasher AZ, Herttuala SY (2007). Progress and prospects: gene therapy clinical trials (part 1) (vol 14, pg 1439, 2007). GENE THER, 14(24), 1754 - 1754. doi:10.1038/sj.gt.3303075
    • Qasim W, Gaspar HB, Thrasher AJ (2007). Update on clinical gene therapy in childhood.. Arch Dis Child, 92(11), 1028 - 1031. doi:10.1136/adc.2006.108787
    • Marshall CJ, Sinclair JC, Thrasher AJ, Kinnon C (2007). Bone morphogenetic protein 4 modulates c-Kit expression and differentiation potential in murine embryonic aorta-gonad-mesonephros haematopoiesis in vitro. British Journal of Haematology, 139(2), 321 - 330. doi:10.1111/j.1365-2141.2007.06795.x
    • Rahim A, Ward N, Waddington S, Elston K, Cooper J, Philpott N, Anderson P, Thrasher A, Raivich G (2007). Cell-specific gene delivery to the adult and in utero CNS using pseudotyped non-integrating lentivirus.
    • Apolonia L, Waddington S, Fernandes C, Ward N, Bouma G, Thrasher A, Collins MK, Philpott N (2007). Long-term expression from non-integrating lentiviral vectors in muscle.
    • Bartholomae C, Balaggan K, Yanez-Munoz RJ, Howe SJ, MacNeil A, Ali R, Thrasher A, von Kalle C, Schmidt M (2007). Nearly random lentiviral integration site patterns observed in postmitotic rodent cells.
    • Ward N, Apolonia L, Fernandes C, Waddington S, Cochrane M, Kinnon C, Thrasher A, Nathwani AC, Philpott N (2007). Non-integrating lentiviral vector pseudotypes for safer gene therapy.
    • Alexander BL, Ali RR, Alton EW, Bainbridge JW, Braun S, Cheng SH, Flotte TR, Gaspar HB, Grez M, Griesenbach U, Kaplitt MG, Ott MG, Seger R, Simons M, Thrasher AJ, Thrasher AZ, Yla-Herttuala S (2007). Progress and prospects: gene therapy clinical trials (part 1). Gene Ther., 14(20), 1439 - 1447.
    • Grez M, Ott MG, Stein S, Schultze-Strasser S, Jauch A, Burwinkel B, Preiss C, Kramer R, Schwarzwaelder K, Siler U, Schmidt M, von Kalle C, Thrasher A, Hoelzer D, Seger R (2007). Update on gene therapy for chronic granulomatous disease.
    • Moulding DA, Blundell MP, Spiller DG, White MR, Cory GO, Calle Y, Kempski H, Sinclair J, Ancliff P, Kinnon C, Jones GE, Thrasher AJ (2007). Unregulated actin polymerization by WASp causes defects of mitosis and cytokinesis in X-linked neutropenia. Journal of Experimental Medicine, 204(9), 2213 - 2224. doi:10.1084/jem.20062324
    • Bacchelli C, Buckridge S, Thrasher AJ, Gaspar HB (2007). Translational mini-review series on immunodeficiency: molecular defects in common variable immunodeficiency. Clin Exp Immunol, 149(3), 401 - 409.
    • Bacchelli C, Buckridge S, Thrasher AJ, Gaspar HB (2007). Translational mini-review series on immunodeficiency: molecular defects in common variable immunodeficiency.. Clin Exp Immunol, 149(3), 401 - 409. doi:10.1111/j.1365-2249.2007.03461.x
    • Apolonia L, Waddington SN, Fernandes C, Ward NJ, Bouma G, Blundell MP, Thrasher AJ, Collins MK, Philpott NJ (2007). Stable Gene Transfer to Muscle Using Non-integrating Lentiviral Vectors. Mol Ther., , - .
    • Schwarzwaelder K, Howe SJ, Schmidt M, Brugman M, Deichmann A, Glimm H, Schmidt S, Prinz C, Wissler M, King DJ, Zhang F, Parsley K, Gilmour KC, Sinclair J, Bayford J, Peraj R, de Ridder D, Pike-Ovezet K, Wagemaker G, Kinnon C, Abel U, Staal F, Gaspar HB, Thrasher AJ, von Kalle C (2007). Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo.. Journal of Clinical Investigation, 117(8), 2241 - 2249. doi:10.1172/JCI31661
    • Deichmann A, Hacein-Bey-Abina S, Schmidt M, Garrigue A, Brugman MH, Hu J, Glimm H, Gyapay G, Prum B, Fraser CC, Fischer N, Schwarzwaelder K, Siegler ML, de Ridder D, Pike-Overzet K, Howe SJ, Thrasher AJ, Wagemaker G, Abel U, Staal FJ, Delabesse E, Villeval JL, Aronow B, Hue C, Prinz C, Wissler M, Klanke C, Weissenbach J, Alexander I, Fischer A, von Kalle C, Cavazzana-Calvo M (2007). Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. J Clin Invest, 117(8), 2225 - 2232.
    • Barker SE, Grosse SM, Siapati EK, Kritz A, Kinnon C, Thrasher AJ, Hart SL (2007). Immunotherapy for neuroblastoma using syngeneic fibroblasts transfected with IL-2 and IL-12. Br J Cancer, 97(2), 210 - 217.
    • Buckley S, Howe S, Sheard V, Anson D, Parsons D, Thrasher A, Coutelle C, Waddington S, McKay T (2007). Significant airway transduction after in utero administration of a gp64 pseudotyped lentiviral vector. doi:10.1002/jgm
    • Philpott NJ, Thrasher AJ (2007). Use of nonintegrating lentiviral vectors for gene therapy.. Hum Gene Ther, 18(6), 483 - 489. doi:10.1089/hum.2007.013
    • Pike-Overzet K, de Ridder D, Weerkamp F, Baert MR, Verstegen MM, Brugman MH, Howe SJ, Reinders MJ, Thrasher AJ, Wagemaker G, van Dongen JJ, Staal FJ (2007). Ectopic retroviral expression of LMO2, but not IL2Rgamma, blocks human T-cell development from CD34+ cells: implications for leukemogenesis in gene therapy. Leukemia, 21(4), 754 - 763.
    • Siapati EK, Bigger BW, Kashofer K, Themis M, Thrasher AJ, Bonnet D (2007). Murine leukemia following irradiation conditioning for transplantation of lentivirally-modified hematopoietic stem cells. Eur J Haematol, 78(4), 303 - 313.
    • Charrier S, Dupre L, Scaramuzza S, Jeanson-Leh L, Blundell MP, Danos O, Cattaneo F, Aiuti A, Eckenberg R, Thrasher AJ, Roncarolo MG, Galy A (2007). Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients. Gene Ther., 14(5), 415 - 428.
    • Himoudi N, Nabarro S, Yan M, Gilmour K, Thrasher AJ, Anderson J (2007). Development of anti-PAX3 immune responses; a target for cancer immunotherapy. Cancer Immunology, Immunotherapy, , - .
    • Qasim W, Mackey T, Sinclair J, Chatziandreou I, Kinnon C, Thrasher AJ, Gaspar HB (2007). Lentiviral vectors for T-cell suicide gene therapy: preservation of T-cell effector function after cytokine-mediated transduction. Molecular Therapy, 15(2), 355 - 360. doi:10.1038/sj.mt.6300042
    • Burns S, Hardy S, Klein N, Thrasher A (2007). CD18 is required for normal podosome formation during adhesion and migration of human dendritic cells.
    • Laurie KL, Blundell MP, Baxendale HE, Howe SJ, Sinclair J, Qasim W, Brunsberg U, Thrasher AJ, Holmdahl R, Gustafsson K (2007). Cell-specific and efficient expression in mouse and human B cells by a novel hybrid immunoglobulin promoter in a lentiviral vector. Gene Therapy, 14(23), 1623 - 1631. doi:10.1038/sj.gt.3303021
    • Baum C, Schambach A, Modlich U, Thrasher A (2007). Gentherapie der SCID-X1. Bundesgesundheitsblatt - Gesundheitsforschung - Gesundheitsschutz, 50(12), 1507 - 1517. doi:10.1007/s00103-007-0385-5
    • Cattoglio C, Facchini G, Sartori D, Antonelli A, Miccio A, Cassani B, Schmidt M, von Kalle C, Howe S, Thrasher AJ, Aiuti A, Ferrari G, Recchia A, Mavilio F (2007). Hot spots of retroviral integration in human CD34+ hematopoietic cells. Blood, 110(6), 1770 - 1778.
    • Barker SE, Grosse S, Siapati KE, Kritz A, Kinnon C, Thrasher AJ, Hart SL (2007). Immunotherapy for Neuroblastoma Delivered by Syngeneic Fibroblasts Transfected with IL-2 and IL-12.. British Journal of Cancer, 97(2), 210 - 217. doi:10.1038/sj.bjc.6603857
    • Zhang F, Thornhill SI, Howe SJ, Ulaganathan M, Schambach A, Sinclair J, Kinnon C, Gaspar HB, Antoniou M, Thrasher AJ (2007). Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells. Blood, 110(5), 1448 - 1457.
    • Abel U, Deichmann A, Bartholomae C, Schwarzwaelder K, Glimm H, Howe S, Thrasher A, Garrigue A, Hacein-Bey-Abina S, Cavazzana-Calvo M, Fischer A, Jaeger D, von Kalle C, Schmidt M (2007). Real-time definition of non-randomness in the distribution of genomic events.. PLoS ONE, 2(6), e570 - .
    • Salzer U, Birmelin J, Bacchelli C, Witte T, Buchegger-Podbielski U, Buckridge S, Rzepka R, Gaspar HB, Thrasher AJ, Schmidt RE, Melchers I, Grimbacher B (2007). Sequence analysis of TNFRSF13b, encoding TACI in patients with Systemic Lupus Erythematosus. Journal of Clinical Immunology, 27(4), 372 - 377.
    • Himoudi N, Nabarro S, Yan M, Gilmour K, Thrasher AJ, Anderson J (2007). Development of anti-PAX3 immune responses; a target for cancer immunotherapy.. Cancer Immunol Immunother, 56(9), 1381 - 1395. doi:10.1007/s00262-007-0294-3

    2006

    • Yanez-Munoz RJ, Balaggan KS, MacNeil A, Howe SJ, Schmidt M, Smith AJ, Buch P, MacLaren RE, Anderson PN, Barker SE, Duran Y, Bartholomae C, Von Kalle C, Heckenlively JR, Kinnon C, Ali RR, Thrasher AJ (2006). Effective gene therapy with non-integrating lentiviral vectors. Nature Medicine, 12(3), 348 - 353. doi:10.1038/nm1365
    • Broderick CA, Smith AJ, Balaggan KS, Georgiadis A, Buch PK, Trittibach PC, Barker SE, Ali RR, Sarra G-M, Thrasher AJ, Dick AD (2006). Erratum: "Local administration of an adeno-associated viral vector expressing IL-10 reduces monocyte infiltration and subsequent photoreceptor damage during experimental autoimmune uveitis" (Molecular Therapy (2005) vol. 12 (369-373) 10.1016/j.ymthe.2005.03.018). Molecular Therapy, 13(4), 829 - 829. doi:10.1016/j.ymthe.2005.09.013
    • Broderick CA, Smith AJ, Balaggan KS, Georgiadis A, Buch PK, Trittibach PC, Barker SE, Sarra GM, Thrasher AJ, Dick AD, Ali RR (2006). Local administration of an adeno-associated viral vector expressing IL-10 reduces monocyte infiltration and subsequent photoreceptor damage during experimental autoimmune uveitis (vol 12, pg 369, 2005). Molecular Therapy, 13(4), 829 - 829.
    • Bigger BW, Siapati EK, Mistry A, Waddington SN, Nivsarkar MS, Jacobs L, Perrett R, Holder MV, Ridler C, Kemball-Cook G, Ali RR, Forbes SJ, Coutelle C, Wright N, Alison M, Thrasher AJ, Bonnet D, Themis M (2006). Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX. Gene Therapy, 13(2), 117 - 126.
    • Chou H-C, Anton LM, Holt M, Curcio C, Lanzardo S, Worth A, Burns S, Thrasher AJ, Jones GE, Calle Y (2006). WIP regulates the stability and localisation of WASP to podosomes in migrating dendritic cells. Current Biology, 16, 2337 - 2344. doi:10.1016/j.cub.2006.10.037
    • Fabes J, Anderson P, Yanez-Munoz RJ, Thrasher A, Brennan C, Bolsover S (2006). Accumulation of the inhibitory receptor EphA4 may prevent regeneration of corticospinal tract axons following lesion. European Journal of Neuroscience, 23(7), 1721 - 1730.
    • Cavazzana-Calvo M, Hacein-Bey-Abina S, Thrasher AJ, Leboulch P, Fischer A (2006). Correction of genetic blood defects by gene transfer. Principles of Molecular Medicine, , 854 - 861. doi:10.1007/978-1-59259-963-9_88
    • Zhang F, Antoniou M, Thrasher AJ (2006). Lentiviral vectors carrying a novel UCOE promoter provide reducible and stable transgene expression.
    • Chandra A, Webster D, Gilmour KC, Kumararatne DS, Thrasher A (2006). Suspected atypical common gamma chain severe combined immunodeficiency. Journal of Allergy and Clinical Immunology, 117(2), S288 - S288.
    • Bartholomae CC, Deichmann A, Schmidt M, Yanez-Munoz RJ, Howe SJ, Schmidt S, Prinz C, Thrasher AJ, von Kalle C (2006). Non-random integration and clone selection by lentiviral SIN-LTR vectors..
    • Booth C, Ribeil JA, Audat F, Dal Cortivo L, Veys PA, Thrasher AJ, Davies EG, Lefrere F, Fischer A, Cavazzana-Calvo M, Gaspar HB (2006). CD34 stem cell top-ups without conditioning after initial haematopoietic stem cell transplantation for correction of incomplete haematopoietic and immunological recovery in severe congenital immunodeficiencies. Br J Haematol, 135(4), 533 - 537.
    • Charrier S, Dupre L, Scaramuzza S, Jeanson-Leh L, Blundell MP, Danos O, Cattaneo F, Aiuti A, Eckenberg R, Thrasher AJ, Roncarolo MG, Galy A (2006). Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients. Gene Ther., , - .
    • Ancliff PJ, Blundell MP, Cory GO, Calle Y, Worth A, Kempski H, Burns S, Jones GE, Sinclair J, Kinnon C, Hann IM, Gale RE, Linch DC, Thrasher AJ (2006). Two novel activating mutations in the Wiskott-Aldrich syndrome protein result in congenital neutropenia. Blood, 108(7), 2182 - 2189. doi:10.1182/blood-2006-01-010249
    • Ancliff PJ, Blundell MP, Cory GO, Calle Y, Worth A, Kempski H, Burns S, Jones GE, Sinclair J, Kinnon C, Hann IM, Gale RE, Linch DC, Thrasher AJ (2006). Two novel activating mutations in the Wiskott-Aldrich syndrome protein result in congenital neutropenia.. Blood, 108(7), 2182 - 2189. doi:10.1182/blood-2006-01-010249
    • Pike-Overzet K, de Ridder D, Weerkamp F, Baert MR, Verstegen MM, Brugman MH, Howe SJ, Reinders MJ, Thrasher AJ, Wagemaker G, van Dongen JJ, Staal FJ (2006). Gene therapy: is IL2RG oncogenic in T-cell development?. Nature, 443(7109), E5 - E7.
    • Thrasher AJ, Gaspar HB, Baum C, Modlich U, Schambach A, Candotti F, Otsu M, Sorrentino B, Scobie L, Cameron E, Blyth K, Neil J, Abina SH, Cavazzana-Calvo M, Fischer A (2006). Gene therapy: X-SCID transgene leukaemogenicity. Nature, 443(7109), E5 - E6.
    • Apolonia L, Waddington S, Collins M, Thrasher A, Philpott N (2006). Development of Non-Integrating and Site-Specifically Integrating Lentiviral Vectors. MOLECULAR THERAPY, 13, S177 - S177.
    • Pike-Overzet K, de Ridder D, Weerkamp F, Baert MRM, Verstegen MM, Howe SJ, Thrasher AJ, Wagemaker G, van Dongen JJM, Staal FJT (2006). Ectopic Retroviral Expression of LMO2, but Not IL2R gamma, Blocks Human T-Cell Development from CD34+Progenitor Cells: Implications for Gene Therapy. MOLECULAR THERAPY, 13, S133 - S133.
    • Gaspar HB, Bjorkegren E, Parsley K, Gilmour KC, King D, Sinclair J, Zhang F, Giannakopoulos A, Adams S, Fairbanks LD, Gaspar J, Henderson L, Xu-Bayford JH, Davies EG, Veys PA, Kinnon C, Thrasher AJ (2006). Successful Reconstitution of Immunity in ADA-SCID by Stem Cell Gene Therapy Following Cessation of PEG-ADA and Use of Mild Preconditioning. Mol Ther., 14(4), 505 - 513.
    • Sabri S, Foudi A, Boukour S, Franc B, Charrier S, Jandrot-Perrus M, Farndale RW, Jalil A, Blundell MP, Cramer EM, Louache F, Debili N, Thrasher AJ, Vainchenker W (2006). Deficiency in the Wiskott-Aldrich protein induces premature proplatelet formation and platelet production in the bone marrow compartment. Blood, 108(1), 134 - 140.
    • Calle Y, Carragher NO, Thrasher AJ, Jones GE (2006). Inhibition of calpain stabilises podosomes and impairs dendritic cell motility. J CELL SCI, 119(11), 2375 - 2385. doi:10.1242/jcs.02939
    • Qasim W, Thrasher AJ (2006). RISC control for gene therapy. NAT BIOTECHNOL, 24(6), 661 - 662.
    • Olivier A, Jeanson-Leh L, Bouma G, Compagno D, Blondeau J, Seye K, Charrier S, Burns S, Thrasher AJ, Danos O, Vainchenker W, Galy A (2006). A Partial Down-regulation of WASP Is Sufficient to Inhibit Podosome Formation in Dendritic Cells. Molecular Therapy, 13(4), 729 - 737. doi:10.1016/j.ymthe.2005.11.003
    • Worth A, Thrasher AJ, Gaspar HB (2006). Autoimmune lymphoproliferative syndrome: molecular basis of disease and clinical phenotype. Br J Haematol, 133(2), 124 - 140.
    • Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U, Glimm H, Kuhlcke K, Schilz A, Kunkel H, Naundorf S, Brinkmann A, Deichmann A, Fischer M, Ball C, Pilz I, Dunbar C, Du Y, Jenkins NA, Copeland NG, Luthi U, Hassan M, Thrasher AJ, Hoelzer D, von Kalle C, Seger R, Grez M (2006). Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nature Medicine, 12(4), 401 - 409. doi:10.1038/nm1393
    • Calle Y, Burns S, Thrasher AJ, Jones GE (2006). The leukocyte podosome. doi:10.1016/j.ejcb.2005.09.003
    • Ochs HD, Thrasher AJ (2006). The Wiskott-Aldrich syndrome. J ALLERGY CLIN IMMUN, 117(4), 725 - 738. doi:10.1016/j.jaci.2006.02.005
    • Howe SJ, Schmidt M, Schwarzwaelder K, Deichmann A, Glimm H, Parsley K, Gilmour HB, Gaspar HB, Kinnon C, Thrasher AJ, Howe AJ (2006). Analysis of retroviralintegration sites in a SCID-X1 gene therapy trial.
    • Booth C, Ribeil JA, Audat F, Dal Cortivo L, Veys PA, Thrasher AJ, Fischer A, Cavazzana-Calvo M, Gaspar HB (2006). CD34+stem cell top-ups without conditioning after initial HSCT for correction of incomplete haematopoietic and immunological recovery in severe congenital immunodeficiencies. Bone Marrow Transplantation, 37, S135 - S135.
    • Titman P, Pink E, Skucek E, Melvin D, Gaspar J, Jones A, Davies G, Thrasher A, Veys P, Gaspar HB (2006). Cognitive and behavioural outcome following haematopoietic stem cell transplantation for severe congenital immunodeficiency. Bone Marrow Transplantation, 37, S59 - S60.
    • Weber MA, Malone M, Thrasher A, Gaspar HB, Sebire NJ (2006). Cutaneous graft versus host disease-like histopathological features following gene therapy.
    • Apolonia L, Waddington S, Thrasher AJ, Collins M, Philpott N (2006). Development of non-integrating lentiviral vectors.
    • Themis M, Waddington SN, Al-Allaf F, Gregory LG, Nivsarkar M, Holder MV, Buckley SMK, Dighe N, Ruthe AT, Bigger B, Rahim A, Coutelle C, Schmidt M, von Kalle C, Wang Y, Mistry A, Thrasher AJ, Nguyen TH, Trono D (2006). Erratum: "Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice" (Molecular Therapy (2005) vol. 12 (763-771) 10.1016/j.ymthe.2005.07.358). Molecular Therapy, 13(4), 830 - 830. doi:10.1016/j.ymthe.2005.10.003
    • Thrasher AJ, Candotti F (2006). Hematopoietic stem cell gene therapy. In Jorde LB, Little PFR, Dunn MJ, Subramaniam S (Ed.), Encyclopedia of Genetics, Genomics, Proteomics and Bioinformatics (pp. 759 - 771). : John Wiley & Sons Ltd..
    • Bailey SI, Howe S, Schambach A, Bohne J, Kinnon C, Baum C, Thrasher AJ (2006). Investigating novel retroviral vectors for the treatment of SCID-X1.

    2005

    • Gaspar HB , Bjorkegren E , Parsley K , Gilmour KC , Sinclair J , Zhang F , Fairbanks LD , King D , Davies G , Veys P , Kinnon C , Thrasher AJ (2005). Somatic gene therapy for ADA-SCID following cessation of PEG-ADA and use of a mild conditioning regime.
    • Thrasher AJ (2005). Update on treatment of human SLID by somatic gene therapy. Toxicology, 213(3), 199 - 199.
    • Nabarro S, Himoudi N, Papanastasiou A, Gilmour K, Gibson S, Sebire N, Thrasher A, Blundell MP, Hubank M, Canderan G, Anderson J (2005). Coordinated oncogenic transformation and inhibition of host immune responses by the PAX3-FKHR fusion oncoprotein.. J Exp Med, 202(10), 1399 - 1410. doi:10.1084/jem.20050730
    • Xue SA, Gao LQ, Hart D, Gillmore R, Qasim W, Thrasher A, Apperley J, Engels B, Uckert W, Morris E, Stauss H (2005). Elimination of human leukemia cells in NOD/SCID mice by WT1-TCR gene-transduced human T cells. BLOOD, 106(9), 3062 - 3067. doi:10.1182/blood-2005-01-0146
    • Themis M, Waddington SN, Schmidt M, von Kalle C, Wang YH, Al-Allaf F, Gregory LG, Nivsarkar M, Themis M, Holder MV, Buckley SMK, Dighe N, Ruthe AT, Mistry A, Bigger B, Rahim A, Nguyen TH, Trono D, Thrasher AJ, Coutelle C (2005). Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice. MOL THER, 12(4), 763 - 771. doi:10.1016/j.ymthe.2005.07.358
    • Gaspar HB, Thrasher AJ (2005). Gene therapy for severe combined immunodeficiencies. EXPERT OPIN BIOL THER, 5(9), 1175 - 1182.
    • Broderick CA, Smith AJ, Balaggan KS, Georgarias A, Buch PK, Trittibach PC, Barker SE, Sarra GM, Thrasher AJ, Dick AD, Ali RR (2005). Local administration of an adeno-associated viral vector expressing IL-10 reduces monocyte infiltration and subsequent photoreceptor damage during experimental autoimmune uveitis. MOL THER, 12(2), 369 - 373.
    • Xue S, Gao L, Hart D, Gillmore R, Qasime W, Thrasher A, Uckert W, Morris E, Stauss H (2005). Development of WT1-specific CTL for the treatment of human leukemia through TCR gene transfer.
    • Thrasher AJ, Gaspar HB (2005). Gene therapy in primary immunodeficiencies.. Expert Rev Clin Immunol, 1(2), 239 - 245. doi:10.1586/1744666X.1.2.239
    • MacLaren RE, Ali RR, Thrasher AJ (2005). Risks of gene therapy should be weighed against lack of alternatives for many diseases. BMJ (Clinical Research Ed.), 330(7494), 791 - . doi:10.1136/bmj.330.7494.791-a
    • Tschernutter M, Schlichtenbrede FC, Howe S, Balaggan KS, Munro PM, Bainbridge JW, Thrasher AJ, Smith AJ, Ali RR (2005). Long-term preservation of retinal function in the RCS rat model of retinitis pigmentosa following lentivirus-mediated gene therapy. GENE THER, 12(8), 694 - 701.
    • de Noronha S, Hardy S, Sinclair J, Blundell MP, Strid J, Schulz O, Zwirner J, Jones GE, Katz DR, Kinnon C, Thrasher AJ (2005). Impaired dendritic-cell homing in vivo in the absence of Wiskott-Aldrich syndrome protein.. Blood, 105(4), 1590 - 1597. doi:10.1182/blood-2004-06-2332
    • Thrasher AJ, Hacein-Bey-Abina S, Gaspar HB, Blanche S, Davies EG, Parsley K, Gilmour K, King D, Howe S, Sinclair J, Hue C, Carlier F, von Kalle C, de Saint BG, le Deist F, Fischer A, Cavazzana-Calvo M (2005). Failure of SCID-X1 gene therapy in older patients. Blood, 105(11), 4255 - 4257.
    • Titman PS, Morton L, Skucek E, Melvin D, Gaspar J, Jones AM, Davies EG, Thrasher AJ, Gaspar HB (2005). Cognitive and behavioural outcome following haematopoietic cell transplantation for severe congenital immunodeficiencies. Bone Marrow Transplantation, 35, S277 - S278.
    • Siapati EK, Bonnet D, Bigger BW, Themis M, Miskin J, Chipchase D, Mitrophanous K, Parsley KL, Thrasher AJ (2005). Comparison of HIV- and EIAV-based vectors on their efficiency in transducing murine and human hematopoietic repopulating cells. Molecular Therapy, 12(3), 537 - 546. doi:10.1016/j.ymthe.2005.01.022
    • Yanez RJ, Balaggan KS, Smith AJ, Buch P, Howe S, MacNeilA D, Y A, P N K, C A, R R T, A J (2005). Effective Gene Therapy with Non-Integrating Lentiviral Vectors.
    • Xue SA, Gao LQ, Hart D, Gillmore R, Qasim W, Thrasher A, Apperley J, Engels B, Uckert W, Morris E, Stauss H (2005). Elimination of human leukemia cells in NOD/SCID mice by WT1-TCR gene-transduced human T cells. Blood, 106(9), 3062 - 3067. doi:10.1182/blood-2005-01-0146
    • Charrier S, Dupre L, Blundell M, Scaramuzza S, Jeanson L, Vainchenker W, Danos O, Roncarolo MG, Thrasher A, Galy A (2005). Functional correction of lymphocytes and dendritic cells of patients with WAS or XLT, using lentiviral vectors targeting the expression of WAS protein to hematopoietic cells. Blood, 106(11), 852A - 852A.
    • Thrasher AJ (2005). Gene Therapy - Great Expectations?. Medical Journal of Australia, 182(9), 440 - 441.
    • Thrasher AJ, Gaspar HB (2005). Gene therapy for lympho-hematopoietic disorders. Current Hematology Reports, 4(4), 305 - 309.
    • Bailey SI, Howe S, Schambach A, Bohne J, Russell DW, Baum C, Thrasher AJ (2005). Investigating novel retroviral vectors for the treatment of SCID-X1.
    • Blundell MP, Charrier S, Kinnon C, Galy A, Thrasher AJ (2005). Lentiviral gene therapy for Wiskotot Aldrich Syndrome.
    • Martin F, Toscano MG, Blundell M, Frecha C, Srivastava GK, Santamaria M, Thrasher AJ, Molina IJ (2005). Lentiviral vectors transcriptionally targeted to hematopoietic cells by WASP gene proximal promoter sequences. Gene Therapy, 12(8), 715 - 723.
    • Themis M, Waddington SN, Schmidt M, Von Kalle C, Wang YH, Al Allaf F, Gregory LG, Nivsarkar M, Themis M, Holder MV, Buckley SMK, Dighe N, Ruthe AT, Mistry A, Bigger B, Rahim A, Nguyen TH, Trono D, Thrasher AJ, Coutelle C (2005). Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice. Molecular Therapy, 12(4), 763 - 771.
    • Gaspar HB, Bjorkegren E, Parsley K, Gilmour KC, Sinclair J, Zhang F, Fairbanks L, King D, Davies EG, Veys P, Kinnon C, Thrasher AJ (2005). Somatic Gene Therapy for ADA-SCID following cessation of PEG-ADA and use of a mild conditioning regime.
    • Gaspar HB, Bjorkegren E, Parsley K, Gilmour KC, Sinclair J, Zhang F, Fairbanks LD, King D, Davies G, Veys P, Kinnon C, Thrasher AJ (2005). Somatic gene therapy for ADA-SCID following cessation of PEG-ADA and use of a mild conditioning regime.
    • Qasim W, Gaspar HB, Thrasher AJ (2005). T cell suicide gene therapy to aid haematopoietic stem cell transplantation. CURR GENE THER, 5(1), 121 - 132.
    • Brooke NJ, Antonious M, Thrasher AJ (2005). To be announced.
    • Parsley KL, Gaspar HB, Howe S, King D, Gilmour KG, Zhang F, Sinclair J, Von Kalle C, Ali RR, Kinnon C, Thrasher AJ (2005). Update on treatment of human SCID-X1 by somatic gene therapy using a pseudotyped gammaretroviral vector.
    • Nabarro S, Himoudi N, Papanastasiou A, Gilmour K, Gibson S, Sebire N, Thrasher A, Blundell MP, Hubank M, Canderan G, Anderson J (2005). Coordinated oncogenic transformation and inhibition of host immune responses by the PAX3-FKHR fusion oncoprotein. Journal of Experimental Medicine, 202(10), 1399 - 1410. doi:10.1084/jem.20050730

    2004

    • Gaspar HB, Harwood C, Leigh I, Thrasher A (2004). Chronic cutaneous papillomavirus disease after haematopoietic stem-cell transplantation in patients with severe combined immune deficiency.
    • Titman P, Pink L, Melvin D, Gaspar J, Jones AM, Davies EG, Thrasher AJ, Gaspar HB (2004). Cognitive and behavioiural outcome following haematopoietic stem cell transplantation for severe congenital immunodeficiencies.
    • Burns S, Thrasher A (2004). Dendritic Cells: The bare bones of immunity. Current Biology, 14(22), R965 - R967.
    • Gaspar HB, Parsley KL, Howe S, King D, Gilmour KC, Sinclair J, Brouns G, Schmidt M, Von Kalle C, Barington T, Jakobsen MA, Christensen HO, Al Ghonaium A, White HN, Smith JL, Levinsky RJ, Ali RR, Kinnon C, Thrasher AJ (2004). Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. The Lancet, 364(9452), 2181 - 2187. doi:10.1016/S0140-6736(04)17590-9
    • Thrasher AJ (2004). Haematological trials for monogenic disorders; lessons to be learned. The Journal of Gene Medicine, 6(9), 43 - .
    • Bigger BW, Siapati KE, Mistry A, Waddington SN, Nivsarkar MS, Holder MV, Ridler C, Kemball-Cook G, Ali RR, Forbes S, Coutelle C, Alison M, Wright N, Thrasher AJ, Bonnet D, Themis M (2004). Induction of immune tolerance and partial phenotypic correction of haermophiliac mice by lentiviral gene transfer of human factor IX to haematopoietic stem cells. The Journal of Gene Medicine, 6(9), 38 - .
    • Balaggan KS, Mistry A, Georgiadis T, Broderick C, Bainbridge J, Thrasher AJ, Ali RR (2004). Inhibition of experimental choroidal neovascularisation by lentivirus mediated delivery of pigment epithelium derived factor. Investigative Ophthalmology and Visual Science, 45, 3705 - .
    • Assari T, Goldblatt D, Thrasher A (2004). Investigating mechanisms of inflammatory resolution in chronic gramulomatous disease.
    • Qasim W, Chatziandreou I, Thrasher A, Gaspar H (2004). Lentiviral vector and suicide gene systems for T-cell therapies. Bone Marrow Transplantation, 33, S22 - S22.
    • Fischer A, Hacein-Bey Abina S, Cavazzana-Calvo M, Thrasher A, Von Kalle C, Stone BD, Rabbitts TH, McCormack MP (2004). LMO2 and gene therapy for severe combined immunodeficiency [7] (multiple letters). New England Journal of Medicine, 350(24), 2526 - 2527. doi:10.1056/NEJM200406103502422
    • Schwarzwaelder K, Schmidt M, Howe S, Prinz C, Wissler M, Schmidt S, Deichmann A, Glimm H, Gaspar HB, Thrasher A, Von Kalle C (2004). Sustained polyclonal hematopoietic repopulation after successful SCID-X1 gene therapy by means of a non random integrating pseudotyped gammaretrovector.
    • Nabarro S, Thrasher A, Kempski H, Anderson J (2004). The Generation of Immunostimulatory Dendritic Cells from the Malignant Clone in Patients with Juvenile Myelomonocytic Leukemia. Leukemia, 17, 1910 - 1912.
    • Gaspar HB, Parsley K, Howe S, King D, Gilmour KC, Sinclair J, White H, Smith J, Barington T, Jacobsen MJ, Christensen HO, Brouns G, AlGhonaim A, Schmidt M, Von Kalle C, Ali RR, Kinnon C, Thrasher AJ (2004). Successful gene therapy of SCID-X1 using a pseudotyped gammaretroviral vector. The Lancet, 364(9452), 2181 - 2187. doi:10.1016/S0140-6736(04)17590-9
    • Burns S, Cory GO, Vainchenker W, Thrasher AJ (2004). Mechanisms of WASp-mediated hematologic and immunologic disease. Blood, 104(12), 3454 - 3462.
    • Waddington SN, Nivsarkar MS, Mistry AR, Buckley SM, Kemball-Cook G, Mosley KL, Mitrophanous K, Radcliffe P, Holder MV, Brittan M, Georgiadis A, Al-Allaf F, Bigger BW, Gregory LG, Cook HT, Ali RR, Thrasher A, Tuddenham EG, Themis M, Coutelle C (2004). Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy.. Blood, 104(9), 2714 - 2721. doi:10.1182/blood-2004-02-0627
    • Calle Y, Chou HC, Thrasher AJ, Jones GE (2004). Wiskott-Aldrich syndrome protein and the cytoskeletal dynamics of dendritic cells. J PATHOL, 204(4), 460 - 469.
    • Martin F, Toscano MG, Blundell M, Frecha C, Srivastava G, Santamaria M, Thrasher AJ, Molina IJ (2004). Lentiviral vectors transcriptionally targeted to hematopoietic cells by WAS gene proximal promoter sequences restore Wiskott-Aldrich Syndrome defects.
    • Gaspar H, Harwood , C , Leigh , I , Thrasher , A J (2004). Severe cutaneous papillomavirus disease after haematopoietic stem-cell transplantation in patients with severe combined immunodeficiency. British Journal of Haematology, 127(2), 232 - 233.
    • Waddington SN, Nivsarkar M, Mistry A, Buckley SMK, Kemball-Cook G, Mosley KL, Mitrophanous K, Radcliffe P, Holder M, Brittan M, Georgiadis T, Al-Allaf F, Bigger B, Gregory L, Cook HT, Ali RR, Thrasher A, Tuddenham EGD, Themis M, Coutelle C (2004). Permanent phenotypic correction of Haemophilia B in immunocompetent mice by prenatal gene therapy.
    • Qasim W, Gaspar HB, Thrasher AJ (2004). Gene therapy for severe combined immune deficiency. EXPERT REV MOL MED, 2004, 1 - 15.
    • Fischer A, Abina SH, Thrasher A, Von Kalle C, Cavazzana-Calvo M (2004). LMO2 and gene therapy for severe combined immunodeficiency. New England Journal of Medicine, 350(24), 2526 - 2527.
    • Schwickerath O, Brouns G, Thrasher A, Kinnon C, Roes J, Casimir C (2004). Enhancer-deleted retroviral vectors restore high levels of superoxide generation in a mouse model of CGD. The Journal of Gene Medicine, 6(6), 603 - 615.
    • Sharifi R, Sinclair J, Gilmour KC, Arkwright PD, Kinnon C, Thrasher AJ, Gaspar HB (2004). SAP mediates specific cytotoxic T-cell functions in X-linked lymphoproliferative disease. Blood, 103(10), 3821 - 3827. doi:10.1182/blood-2003-09-3359
    • Booth MJ, Mistry A, Li X, Thrasher A, Coffin RS (2004). Transfection-free and scalable recombinant AAV vector production using HSV/AAV hybrids. Gene Therapy, 11(10), 829 - 837.
    • Booth MJ, Mistry A, Li X, Thrasher A, Coffin RS (2004). Transfection-free and scalable recombinant AAV vector production using HSV/AAV hybrids.. Gene Ther, 11(10), 829 - 837. doi:10.1038/sj.gt.3302226
    • Ahmed F, Ings SJ, Pizzey AR, Blundell MP, Thrasher AJ, Ye HT, Fahey A, Linch DC, Yong KL (2004). Impaired bone marrow homing of cytokine-activated CD34+ cells in the NOD/SCID model. Blood, 103(6), 2079 - 2087. doi:10.1182/blood-2003-06-1770
    • Burns SO, Hardy SJ, Buddle J, Yong KL, Jones GE, Thrasher AJ (2004). Maturation of DC is associated with changes in motile characteristics and adherence. Cell Motility and the Cytoskeleton, 57, 118 - 132.
    • Cavazzana-Calvo M, Thrasher A, Mavilio F (2004). The future of gene therapy. Nature, 427(6977), 779 - 781.
    • Thrasher AJ, Edwards RG (2004). Averting abnormal inheritance: potential of gene therapy and preimplantation diagnosis. REPROD BIOMED ONLINE, 8(1), 99 - 106.
    • de Noronha S, Hardy S, Sinclair J, Blundell MP, Strid J, Schulz O, Zwirner J, Jones GE, Katz DR, Kinnon C, Thrasher AJ (2004). Impaired dendritic cell homing in vivo in the absence of Wiskott-Aldrich syndrome protein. Blood, 105(4), 1590 - 1597. doi:10.1182/blood-2004-06-2332
    • Calle Y, Jones GE, Jagger C, Fuller K, Blundell MP, Chow J, Chambers T, Thrasher AJ (2004). WASp deficiency in mice results in failure to form osteoclast sealing zones and defects in bone resorption. Blood, 103(9), 3552 - 3561.
    • Gennery AR, Thrasher AJ (2004). Gene therapy for severe combined immunodeficiency. Current Anaesthesia and Critical Care, 15(1), 3 - 9. doi:10.1016/j.cacc.2004.01.008
    • Schlichtenbrede FC, Smith AJ, Bainbridge JW, Thrasher AJ, Salt TE, Ali RR (2004). Improvement of neuronal visual responses in the superior colliculus in Prph2(Rd2/Rd2) mice following gene therapy.. Molecular and Cellular Neuroscience, 25(1), 103 - 110. doi:10.1016/j.mcn.2003.09.020
    • Blundell M, Ancliff P, Cory G, Jones G, de Botton S, Vainchanker W, Thrasher AJ (2004). Unregulated activation of the actin cytoskeleton through WASp promoters cell death and genomic instability.
    • Gaspar HB, Parsley K, Howe S, Gilmour K, Sinclair J, Barington A, Algonaim A, Davies G, Kinnon C, Thrasher A (2004). Update on treatment of human SCID-X1 by somatic gene therapy using a pseudotyped gammaretroviral vector.
    • Hubbard VG, Breuer J, Leigh IM, Thrasher A, Harwood CA (2004). Viral warts following bone marrow transplantation for severe combined immunodeficiency harbour potentially oncogenic human papillomavirus types. British Journal of Dermatology, 151, 19 - 19.
    • Westerberg L, Larsson M, Hardy SJ, Fernandez C, Thrasher AJ, Severinson E (2004). Wiskott-Aldrich syndrome protein deficiency leads to reduced B-cell adhesion, migration, and homing, and a delayed humoral immune response. Blood, 105(3), 1144 - 1152.

    2003

    • Nabarro S, Thrasher AJ, Kempski H, Amrolia P, Anderson J (2003). Generation of immunostimulatory dendritic cells from the malignant clone in patients with juvenile myelomonocytic leukemia. Leukemia, 17(9), 1910 - 1912. doi:10.1038/sj.leu.2403059
    • Bainbridge JBL, Mistry A, Thrasher A, Ali RR (2003). Gene therapy for ocular angiogenesis. Clinical Science, 104(6), 561 - 575.
    • Bainbridge JWB, Mistry A, Schlichtenbrede FC, Smith A, Broderick C, De Alwis M, Georgiadis A, Taylor PM, Squires M, Sethi C, Charteris D, Thrasher AJ, Sargan D, Ali RR (2003). Stable rAAV-mediated transduction of rod and cone photoreceptors in the canine retina. Gene Therapy, 10(16), 1336 - 1344. doi:10.1038/sj.gt.3301990
    • Bainbridge JWB, Mistry A, Binley K, De Alwis M, Thrasher A, Naylor S, Ali RR (2003). Hypoxia-regulated transgene expression in experimental retinal and choroidal neovascularisation. Gene Therapy, 10(12), 1049 - 1054. doi:10.1038/sj.gt.3301945
    • Brown JR, Goldblatt D, Buddle J, Morton L, Thrasher AJ (2003). Diminished production of anti-inflammatory mediators during neutrophil apoptosis and macrophage phagocytosis in chronic granulomatous disease (CGD). Journal of Leukocyte Biology, 73(5), 591 - 599.
    • Marden C, Cunninghame Graham D, Thrasher A, Casimir C (2003). A functional ISRE is required for myeloid transcription of the p47(phox) gene. Biochimica et Biophysica Acta (BBA) - Gene Structure and Expression, 1630(2-3), 117 - 122. doi:10.1016/j.bbaexp.2003.09.005
    • Ahmed F, Ings SJ, Pizzey AR, Blundell MP, Thrasher AJ, Ye HT, Fahey A, Linch DC, Yong KL (2003). Impaired bone marrow homing of cytokine activated CD34+ cells in the NOD/SCID model. BLOOD, , - .
    • Gaspar HB, Howe S, Thrasher AJ (2003). Gene therapy progress and prospects: gene therapy for severe combined immunodeficiency. Gene Therapy, 10(24), 1999 - 2004.
    • Qasim W, King D, Buddle J, Verfuerth S, Kinnon C, Thrasher AJ, Gaspar HB (2003). The impact of retroviral suicide gene transduction procedures on T cells. British Journal of Haematology, 123(4), 712 - 719. doi:10.1046/j.1365-2141.2003.04672.x
    • Smith AJ, Schlichtenbrede FC, Tschernutter M, Bainbridge JW, Thrasher AJ, Ali RR (2003). AAV-Mediated gene transfer slows photoreceptor loss in the RCS rat model of retinitis pigmentosa. Molecular Therapy, 8(2), 188 - 195.
    • Howe S, Thrasher AJ (2003). Gene therapy for inherited immunodeficiencies.. Curr Hematol Rep, 2(4), 328 - 334.
    • Siapati KE, Barker S, Kinnon C, Michalski A, Anderson E, Brickell P, Thrasher AJ, Hart SL (2003). Improved antitumour immunity in murine neuroblastoma using a combination of IL-2 and IL-12.. British Journal of Cancer, 88(10), 1641 - 1648. doi:10.1038/sj.bjc.6600928
    • Westerberg L, Hardy S, Greicius G, Thrasher AJ, Severinson E (2003). B cell and splenic abnormalities in Wiskott-Aldrich syndrome.
    • Schlichtenbrede FC, MacNeil A, Bainbridge JW, Tschernutter M, Thrasher AJ, Smith AJ, Ali RR (2003). Intraocular gene delivery of ciliary neurotrophic factor results in significant loss of retinal function in normal mice and in the Prph2Rd2/Rd2 model of retinal degeneration. Gene Therapy, 10(6), 523 - 527.
    • Thrasher A, Edwards RG (2003). Averting abnormal inheritance: potential of gene therapy and preimplantation diagnosis. Reproductive Biomedicine Online, 8(1), 99 - 106.
    • Uduehi A, Mailhos C, Truman H, Thrasher A, Kinnon C, Hart SL (2003). Enhancement of integrin-mediated transfection of haematopoietic cells with a synthetic vector system. Biotechnology and Applied Biochemistry, 38, 201 - 209.
    • Howe S, Thrasher A (2003). Gene therapy for inherited immunodeficiencies. Current Hematology Reports, 2, 283 - 334.
    • Schlichtenbrede FC, da Cruz L, Stephens C, Smith AJ, Georgiadis A, Thrasher AJ, Bainbridge JW, Seeliger MW, Ali RR (2003). Long-term evaluation of retinal function in Prph2Rd2/Rd2 mice following AAV-mediated gene replacement therapy. The Journal of Gene Medicine, 5(9), 757 - 764.

    2002

    • Bainbridge JWB, Mistry A, De Alwis M, Paleolog E, Baker A, Thrasher AJ, Ali RR (2002). Inhibition of retinal neovascularisation by gene transfer of soluble VEGF receptor sFlt-1. Gene Therapy, 9(5), 320 - 326. doi:10.1038/sj.gt.3301680
    • Gaspar HB, Sharifi R, Gilmour KC, Thrasher AJ (2002). X-linked lymphoproliferative disease: clinical, diagnostic and molecular perspective. British Journal of Haematology, 119(3), 585 - 595. doi:10.1046/j.1365-2141.2002.03851.x
    • Kinnon C, Thrasher AJ (2002). Gene therapy for single gene disorders. In Brooks G (Ed.), Gene Therapy. The use of DNA as a drug (pp. 71 - 85). : Pharmaceutical Press.
    • Mistry AR, De Alwis M, Feudner E, Ali RR, Thrasher AJ (2002). High-titer stocks of adeno-associated virus from replicating amplicons and herpes vectors. Methods in Molecular Medicine, 69, 445 - 460.
    • Hacein-Bey-Abina S, le Deist F, Carlier F, Bouneaud C, Hue C, De Villartay JP, Thrasher AJ, Wulffraat N, Sorensen R, Dupuis-Girod S, Fischer A, Davies EG, Kuis W, Leiva L, Cavazzana-Calvo M (2002). Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N.Engl.J Med, 346(16), 1185 - 1193.
    • Jones L, Flood TJ, McGrogan P, Morton L, Parker L, Goldblatt D, Thrasher A, Cant AJ (2002). Respiratory disease in Chronic Granulomatous Disease.
    • Sharifi GR, Kinnon C, Thrasher A, Gaspar BH (2002). NK cells as new target for gene therapy; Using retroviral vectors as promising delivery system..
    • Sharifi GR, Buddle JOC, Kinonn C, Thrasher A, Gaspar BH (2002). Reconstitution of XLP CTL function by gene transfer; A gene therapy model for XLP..
    • Binley K, Askham Z, Iqball S, Spearman H, Martin L, De Alwis M, Thrasher AJ, Ali RR, Maxwell PH, Kingsman S, Naylor S (2002). Long-term reversal of chronic anemia using a hypoxia-regulated erythropoietin gene therapy. BLOOD, 100(7), 2406 - 2413.
    • Calle Y, Jagger C, Chambers T, Fuller K, Blundell MP, Chow J, Jones GE, Thrasher A (2002). WASp deficiency in mice results in abnormal assembly of podosomes in osteoclasts and defects in bone resorption.
    • Qasim W, Thrasher AJ, Buddle JC, Kinnon C, Black M, Gaspar HB (2002). T cell transduction and suicide with an anhanced mutant thymidine kinase. Gene Therapy, 9(12), 824 - 827. doi:10.1038/sj.gt.3301690
    • Qasim W, Thrasher AJ, Buddle J, Kinnon C, Black ME, Gaspar HB (2002). T cell transduction and suicide with an enhanced mutant thymidine kinase. GENE THER, 9(12), 824 - 827. doi:10.1038/sj/gt/3301690
    • Morgan JE, Gross JG, Pagel CN, Beauchamp JR, Fassati A, Thrasher AJ, Di Santo JP, Fisher IB, Shiwen X, Abraham DJ, Partridge TA (2002). Myogenic cell proliferation and generation of a reversible tumorigenic phenotype are triggered by preirradiation of the recipient site. The Journal of Cell Biology, 157(4), 693 - 702. doi:10.1083/jcb.200108047
    • Demaison C, Parsley K, Brouns G, Scherr M, Battmer K, Kinnon C, Grez M, Thrasher AJ (2002). High-level transduction and gene expression in hematopoietic repopulating cells using a human immunodeficiency virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter. Human Gene Therapy, 13(7), 803 - 813. doi:10.1089/10430340252898984
    • Sarra GM, Stephens C, Schlichtenbrede FC, Bainbridge JW, Thrasher AJ, Luthert PJ, Ali RR (2002). Kinetics of transgene expression in mouse retina following sub-retinal injection of recombinant adeno-associated virus. Vision Research, 42(4), 541 - 549.
    • Thrasher AJ, Segal AW (2002). Chronic granulomatous disease and other phagocytic disorders. In (Ed.), Encyclopaedia of Life Sciences (pp. - ). : .
    • Lucas VS, Gaspar H, Thrasher A, Jones A, Gaspar J, Roberts GJ, Davies EG (2002). Dental development following conditioned bone marrow transplantation in early infancy for primary immunodeficiency disorders.
    • Thrasher AJ, Brown J (2002). Disorders of Phagocyte Function. In Wickramasinghe M (Ed.), Blood and Bone Marrow Pathology. (pp. - ). : Harcourt.
    • Hacein-Bey-Abina S, Le Deist F, De Villartay J-P, Fischer A, Cavazzana-Calvo M, Carlier F, Hue C, Dupuis-Girod S, Bouneaud C, Thrasher AJ, Wulffraat N, Kuis W, Sorensen R, Leiva L, Graham Davies E (2002). Erratum: Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy (New England Journal of Medicine (April 18, 2002) 346 (1185-1193)). New England Journal of Medicine, 347(8), 613 - 613. doi:10.1056/NEJM200208223470814
    • Li JM, Mullen AM, Yun S, Wientjes F B, G T, A J S, A M (2002). Essential role of the NADPH oxidase subunit p47PHOX in endothelial cell superoxide production in response to phorbol ester and tumor necrosis factor-alpha. Circulation Research, 90, 143 - 150.
    • Yong KL, Fahey A, Pahal G, Linch DC, Pizzey A, Thomas NSB, Jauniaux E, Kinnon C, Thrasher AJ (2002). Fetal haemapoietic cells display enhanced migration across endothelium. British Journal of Haematology, 116, 392 - 400. doi:10.1046/j.1365-2141.2002.03273.x
    • Langrish CL, Buddle JC, Thrasher AJ, Goldblatt D (2002). Neonatal dendritic cells are intrinsically biased against Th-1 immune responses. Clinical and Experimental Immunology, 128(1), 118 - 123.
    • Jones GE, Zicha D, Dunn GA, Blundell M, Thrasher AJ (2002). Restoration of podosomes and chemotaxis in Wiskott-Aldrich syndrome macrophages following induced expression of WASp. The International Journal of Biochemistry and Cell Biology, 34, 806 - 815.
    • (2002). WASp in immune-system organisation and function. Nature Reviews Immunology, 2(9), 635 - 646.
    • Bainbridge JWB, Mistry A, De Alwis M, Paleolog E, Baker A, Thrasher AJ, Ali RR (2002). Inhibition of retinal neovascularisation by gene transfer of soluble VEGF receptor sFlt-1. GENE THER, 9(5), 320 - 326. doi:10.1038/sj/gt/3301680

    2001

    • Qasim W, Gilmour KC, Heath S, Ashton E, Cranston T, Thomas A, Finn A, Davies EG, Thrasher AJ, Kinnon C, Jones A, Gaspar HB (2001). Protein assays for diagnosis of Wiskott-Aldrich syndrome and X-linked thrombocytopenia. Br.J Haematol., 113(4), 861 - 865.
    • Feudner E, De Alwis M, Thrasher AJ, Ali RR, Fauser S (2001). Optimization of recombinant adeno-associated virus production using an herpes simplex virus amplicon system. Journal of Virological Methods, 96(2), 97 - 105.
    • Leverrier Y, Lorenzi R, Blundell MP, Brickell P, Kinnon C, Ridley AJ, Thrasher AJ (2001). Cutting edge: the Wiskott-Aldrich syndrome protein is required for efficient phagocytosis of apoptotic cells.. The Journal of Immunology, 166(8), 4831 - 4834.
    • (2001). Genetic incorporation of the SIGYPLP peptide into adenoassociated virus-2 capsids directs efficient and selective gene transfer to the human vascular endothelial cells. Molecular Therapy, 4, 174 - 181.
    • Thrasher A, Goldblatt D (2001). Hematopoietic-cell transplantation for chronic granulomatous disease. New England Journal of Medicine, 345(5), 377 - 378.
    • Sarra G, Stephens C, De Alwis M, Bainbridge J, Munro P, Thrasher AJ, Ali RR (2001). Long term restoration of photoreceptor outer segments in the rds mouse after gene replacement therapy. Investigative Ophthalmology and Visual Science, 42(4), 1878 - .
    • Qasim W, Gilmour KC, Ashton E, Graham Davies E, Thrasher AJ, Jones A, Gaspar HB, Kinnon C, Heath S, Cranston T, Thomas A, Finn A (2001). Protein assays for diagnosis of Wiskott-Aldrich syndrome and X-linked thrombocytopenia. British Journal of Haematology, 113(4), 861 - 865. doi:10.1046/j.1365-2141.2001.02832.x
    • (2001). The embryonic origins of human haematopoiesis. British Journal of Haematology, 112, 838 - 850.
    • Reichel MB, Bainbridge J, Baker D, Thrasher AJ, Bhattacharya SS, Ali RR (2001). An immune response after intraocular administration of an adenoviral vector containing a beta galactosidase reporter gene slows retinal degeneration in the rd mouse. British Journal of Ophthalmology, 85(3), 341 - 344.
    • Patton GS, Brouns G, Thrasher AJ, McClure MO (2001). Transduction of haematopoietic stem cells (HSC's) with human foamy virus (HFV) vectors. AIDS RES HUM RETROV, 17, S58 - S58.
    • Ancliff PJ, Blundell MP, Gale RE, Liesner R, Hann IM, Thrasher AJ, Linch DC (2001). Activating mutations in theWiskott Aldrich syndrome protein may define a sub-group of severe congenital neutropenia (SCN) with specific and unusual laboratory features.. BLOOD, 98(11), 439A - 440A.
    • Ahmed F, Ings SJ, Pizzey A, Fahey A, Blundell M, Hardy SJ, Thrasher A, Linch DC, Yong KL (2001). Cytokine exposure impairs short-term homing of CD34(+) cells in SCID/NOD mice: Influence of cell cycle status.. BLOOD, 98(11), 644A - 644A.
    • (2001). Activating mutations in theWiskott Aldrich syndrome protein may define a sub-group of severe congenital neutropenia (SCN) with specific and unusual laboratory features. Blood, 98(11), 1842 - .
    • (2001). In vivo gene tranfer to the mouse eye using an HIV-based lentiviral vector; efficient long term transduction of corneal endothelium and retinal pigment epithelium. Gene Therapy, 8(21), 1665 - 1668. doi:10.1038/sj.gt.3301574
    • Sarra GM, Stephens C, De Alwis M, Bainbridge JW, Smith AJ, Thrasher AJ, Ali RR (2001). Gene replacement therapy in the retinal degeneration slow (rds) mouse: the effect on retinal degeneration following partial transduction of the retina. Human Molecular Genetics, 10(21), 2353 - 2361.
    • Nicklin SA, Buening H, Dishart KL, De Alwis M, Girod A, Hacker U, Thrasher AJ, Ali RR, Hallek M, Baker AH (2001). Efficient and selective AAV2-mediated gene transfer directed to human vascular endothelial cells. Molecular Therapy, 4(3), 174 - 181.
    • Ali RR, Sarra GM, Stephens CM, de Alwis M, Bainbridge JWB, Thrasher AJ (2001). Gene therapy for inherited retinal degeneration. J MED GENET, 38, S26 - S26.
    • Burns SO, Thrasher AJ, Blundell MP, Machesky L, Jones GE (2001). Configuration of human dendritic cell cytoskeleton by Rho GTPases, the WAS protein, and differentiation. Blood, 98(4), 1142 - 1149. doi:10.1182/blood.V98.4.1142
    • Wright MJ, Wightman LM, Lilley C, De Alwis M, Hart SL, Miller A, Coffin RS, Thrasher A, Latchman DS, Marber MS (2001). In vivo myocardial gene transfer: optimization, evaluation and direct comparison of gene transfer vectors. Basic Research in Cardiology, 96(3), 227 - 236.

    2000

    • Gilmour KC, Cranston T , Jones AM , Davies EG, Goldblatt D , Thrasher AJ , Kinnon C , Nichols KE , Gaspar HB (2000). Diagnosis of X-linked lymphoproliferative disease by analysis of SLAM-associated protein expression.. Eur.J.Immun., 30(6), 1691 - 1697.
    • White H, Thrasher A, Veys P, Kinnon C, Gaspar HB (2000). Intrinsic defects of B cell function in X-linked severe combined immunodeficiency. European Journal of Immunology, 30(3), 732 - 737.
    • Pahal GS, Jauniaux ER, Kinnon C, Thrasher A, Rodeck CH (2000). Normal development of human fetal hematopoiesis between eight and seventeen weeks' gestation. American Journal of Obstetrics and Gynecology, 183, 1029 - 1034.
    • Marshall CJ, Kinnon C, Thrasher AJ (2000). Polarised expression of bone morphogenic protein-4 in the human aorta-gonad-mesonephros region. BLOOD, 96, 1591 - 1593.
    • (2000). Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy. Nature Genetics, 25(3), 306 - 310.
    • Lorenzi R, Brickell P, Katz DR, Kinnon C, Thrasher AJ (2000). Wiskott-Aldrich syndrome protein is necessary for effecient IgG-mediated phagocytosis. BLOOD, 95, 2943 - 2946.
    • Thrasher AJ, Burns S, Lorenzi R, Jones GE (2000). The Wiskott-Aldrich syndrome: disordered actin dynamics in haematopoietic cells. Immunological Reviews, 178, 118 - 128.
    • Demaison C, Brouns G, Blundell MP, Goldman JP, Levinsky RJ, Grez M, Kinnon C, Thrasher AJ (2000). A defined window for efficient gene marking of severe combined immunodeficient-repopulating cells using a gibbon ape leukemia virus pseudotyped retroviral vector. Human Gene Therapy, 11(1), 91 - 100.
    • Demaison C, Brouns G, Blundell MP, Goldman JP, Levinsky RJ, Grez M, Kinnon C, Thrasher AJ (2000). A defined window for efficient gene marking of severe combined immunodeficient-repopulating cells using a gibbon ape leukemia virus-pseudotyped retroviral vector.. Hum Gene Ther, 11(1), 91 - 100. doi:10.1089/10430340050016184
    • Goldblatt D, Thrasher A (2000). Chronic granulomatous disease. Clinical and Experimental Immunology, 122(1), 1 - 9.
    • Hudde T, Rayner SA, De Alwis M, Thrasher AJ, Smith J, Coffin RS, George AJ, Larkin DF (2000). Adeno-associated and herpes simplex viruses as vectors for gene transfer to the corneal endothelium. CORNEA, 19(3), 369 - 373.
    • Thrasher AJ, Kinnon C (2000). The Wiskott-Aldrich syndrome. Clinical and Experimental Immunology, 120, 2 - 9.

    1999

    • Brouns G, Demaison C, Blundell M, Goldman J, Levinsky RJ, Grez M, Kinnon C, Thrasher AJ (1999). Evidence for human myeloid-restricted haematopoietic stem cells. British Journal of Haematology, 105, 284 - .
    • Zhang X, De Alwis M, Hart SL, Fitzke FW, Inglis SC, Boursnell ME, Levinsky RJ, Kinnon C, Ali RR, Thrasher AJ (1999). High-titer recombinant adeno-associated virus production from replicating amplicons and herpes vectors deleted for glycoprotein H.. Hum Gene Ther, 10(15), 2527 - 2537. doi:10.1089/10430349950016861
    • Davies EG, Thrasher A, Lacey K, Harper J (1999). Topical cidofovir for severe molluscum contagiosum. Lancet, 353(9169), 2042 - .
    • Thrasher AJ, Burns S (1999). Wiskott-Aldrich syndrome: a disorder of haematopoietic cytoskeletal regulation.. Microsc Res Tech, 47(2), 107 - 113. doi:10.1002/(SICI)1097-0029(19991015)47:2<107::AID-JEMT3>3.0.CO;2-H
    • Pahal G, Yong K, Fahey A, Thomas NSB, Rodeck C, Thrasher A (1999). Functional characterisation of primitive human haemopoietic cells in foetal blood.. BLOOD, 94(10), 128A - 128A.
    • Blundell MP, Demaison C, Brouns G, Goldman JP, Gaspar HB, Kinnon C, Thrasher AJ, Lazzari L, Sirchia G (1999). Quality of repopulation in nonobese diabetic severe combined immunodeficient mice engrafted with expanded cord blood CD34(+) cells. Blood, 94(9), 3269 - 3270.
    • Goldblatt D, Butcher J, Thrasher AJ, Russell-Eggit I (1999). Ocular findings in chronic granulomatous disease. The Journal of Pediatrics, 134, 780 - 783.
    • Gaspar HB, White HN, Veys PA, Thrasher AJ (1999). Molecular analysis of B cell repertoire development in patients with X-linked severe combined immunodeficiency following haematopoietic stem cell transplantation. Bone Marrow Transplantation, 23, 580 - .
    • Bainbridge J, Stephens C, Zhang Z, Reichel MB, Thrasher AJ, Ali RR (1999). Efficient transduction of photoreceptor cells using high titre rAAV produced from replicating amplicons and gH- herpes vectors. Investigative Ophthalmology and Visual Science, 40, 44747 - .
    • Wright MJ, Wightman L, Lilley C, Coffin R, Marber MS, Latchman DS, De Alwis M, Thrasher A (1999). Differences in timecourse of gene expression following direct intramyocardial injection of recombinant adenovirus, adeno-associated virus and herpes simplex virus. Heart, 81(SUPPL. 1), - .
    • Marshall CJ, Moore R, Thorogood P, Brickell PM, Kinnon C, Thrasher AJ (1999). Detailed characterisation of the human aorta-gonad-mesonephros region reveals morphological polarity resembling a hematopoietic stromal layer. Developmental Dynamics, 215, 139 - 147.
    • Goldblatt D, Butcher J, Thrasher A, Russell-Eggitt IM (1999). Chorioretinal lesions in patients and carriers of chronic granulomatous disease. The Journal of Pediatrics, 134, 780 - 783.

    1998

    • Coffin RS, Thomas SK, Thomas NSB, Lilley CE, Pizzey AR, Griffiths CH, Gibb BJ, Wagstaff MJD, Inges SJ, Binks MH, Chain BM, Thrasher AJ, Rutault K, Latchman DS (1998). Populations of transduced primary human cells can be produced using GFP expressing herpes virus vectors and flow cytometry. Gene Therapy, 5, 718 - 722.
    • Goldman JP, Blundell MP, Lopes L, Kinnon C, Di Santo JP, Thrasher AJ (1998). Enhanced human cell engraftment in mice deficient in RAG2 and the common cytokine receptor gamma chain.. Br J Haematol, 103(2), 335 - 342.
    • Coffin RS, Thomas SK, Thomas NSB, Lilley CE, Pizzey AR, Griffiths CH, Gibb BJ, Wagstaff MJ, Inges SJ, Binks MH, Chain BM, Thrasher AJ, Rutault K, Latchman DS (1998). Pure populations of transduced primary human cells can be produced using GFP expressing herpes virus vectors and flow cytometry. Gene Therapy, 5(5), 718 - 722.
    • Zicha D, Allen WE, Brickell PM, Kinnon C, Dunn GA, Jones GE, Thrasher AJ (1998). Chemotaxis of macrophages is abolished in the Wiskott-Aldrich syndrome. BRIT J HAEMATOL, 101(4), 659 - 665.
    • Binks MH, Jones JE, Brickell PM, Kinnon C, Katz DR, Thrasher AJ (1998). Intrinsic dendritic cell abnormalities in Wiskott-Aldrich syndrome. European Journal of Immunology, 28, 3259 - 3267.
    • Brickell PM, Katz DR, Thrasher AJ (1998). Wiskott-Aldrich syndrome: current research concepts. British Journal of Haematology, 101, 603 - 608.
    • Ali RR, Reichel MB, de Alwis M, Kanuga N, Kinnon C, Levinsky RJ, Hunt DM, Bhattacharya SS, Thrasher AJ (1998). Adeno-associated virus gene transfer to mouse retina. Human Gene Therapy. Human Gene Therapy, 9, 81 - 86.
    • Ali RR, Reichel MB, Baker D, Byrnes AP, Kanuga N, Hunt DM, Bhattacharya SS (1998). Co-injection of adenovirus expressing CTLA4-Ig prolongs adenovirally-mediated LacZ reporter gene expression in the mouse retina.. Gene Therapy, 5(11), 1561 - 1565.
    • Reichel MB, Ali RR, Thrasher AJ, Hunt DM, Bhattacharya SS, Baker D (1998). Immune responses limit adenovirally-mediated gene expression in the adult mouse eye.. Gene Therapy, 5(8), 1038 - 1046.
    • Wright MJ, Wightman L, Lilley C, Coffin R, Marber MS, Latchman DS, Stewart L, Miller A, Thrasher A (1998). In vivo gene transfer to the adult rabbit myocardium: Systematic comparison of models and vectors. Heart, 79(SUPPL. 1), - .
    • Thrasher AJ, Jones G, Kinnon C, Brickell PM, Katz DR (1998). Is Wiskott-Aldrich syndrome a cell trafficking disorder?. Immunology Today, 19, 537 - 539.
    • Thrasher AJ, Binks M, Kinnon C, Katz DR, Brickell PM, Jones GE (1998). Intrinsic dendritic cell abnormalities in Wiskott-Aldrich syndrome.. J LEUKOCYTE BIOL, , 52 - 52.
    • Hart SL, Arancibia-Cárcamo CV, Wolfert MA, Mailhos C, O'Reilly NJ, Ali RR, Coutelle C, George AJ, Harbottle RP, Knight AM, Larkin DF, Levinsky RJ, Seymour LW, Thrasher AJ, Kinnon C (1998). Lipid-mediated enhancement of transfection by a nonviral integrin-targeting vector.. Hum Gene Ther, 9(4), 575 - 585. doi:10.1089/hum.1998.9.4-575
    • Binks M, Jones GE, Brickell PM, Kinnon C, Katz DR, Thrasher AJ (1998). Intrinsic dendritic cell defects in Wiskott-Aldrich syndrome.. MOL IMMUNOL, 35(11-12), 734 - 734.
    • Goldman JP, Blundell MP, Kinnon C, DiSanto JP, Thrasher AJ (1998). Enhanced human cell engraftment in mice deficient in RAG2 and the common cytokine receptor gamma chain. MOL IMMUNOL, 35(11-12), 779 - 779.
    • Wright MJ, Wightman LML, de Alwis M, Lilley C, Stewart L, Hart S, Miller A, Coffin RS, Latchman DS, Thrasher AJ, Marber MS (1998). Intracoronary and direct intramyocardial injection gene transfer to the in vivo rabbit heart: A comparison of viral and non-viral vectors.. CIRCULATION, 98(17), 527 - 527.
    • Ali RR, Reichel MB, De Alwis M, Kanuga N, Kinnon C, Levinsky RJ, Hunt DM, Bhattacharya SS, Thrasher AJ (1998). Adeno-associated virus gene transfer to mouse retina.. Hum Gene Ther, 9(1), 81 - 86. doi:10.1089/hum.1998.9.1-81

    1997

    • Thrasher AJ, Goldman J, de Alwis M, Porter CD, Kinnon C (1997). Gene therapy for primary immunodeficiency.. Biochem Soc Trans, 25(2), 537 - 540.
    • Ali RR, Reichel MB, Thrasher AJ, Kanuga N, Hunt DM, Bhattacharya SS (1997). Gene delivery to the RDS mouse. INVEST OPHTH VIS SCI, 38(4), 1194 - 1194.
    • Thrasher A (1997). Somatic gene therapy.. Br J Hosp Med, 57(7), 351 - .

    1996

    • Ali RR, Reichel MB, Thrasher AP, Kanuga N, Hunt DM, Bhattacharya SS (1996). In vivo gene delivery to the mouse retina. INVEST OPHTH VIS SCI, 37(3), 41 - 41.
    • Ali RR, Reichel MB, Thrasher AJ, Levinsky RJ, Kinnon C, Kanuga N, Hunt DM, Bhattacharya SS (1996). Gene transfer into the mouse retina mediated by an adeno-associated viral vector.. Hum Mol Genet, 5(5), 591 - 594.

    1995

    • Thrasher A, Kinnon C (1995). Gene therapy for primary immunodeficiency.. Gene Ther, 2(9), 601 - 602.
    • Thrasher AJ, de Alwis M, Casimir CM, Kinnon C, Page K, Lebkowski J, Segal AW, Levinsky RJ (1995). Generation of recombinant adeno-associated virus (rAAV) from an adenoviral vector and functional reconstitution of the NADPH-oxidase.. Gene Ther, 2(7), 481 - 485.
    • THRASHER AJ, CASIMIR CM, KINNON C, MORGAN G, SEGAL AW, LEVINSKY RJ (1995). GENE-TRANSFER TO PRIMARY CHRONIC GRANULOMATOUS-DISEASE MONOCYTES. LANCET, 346(8967), 92 - 93.
    • Chetty M, Thrasher AJ, Abo A, Casimir CM (1995). Low NADPH oxidase activity in Epstein-Barr-virus-immortalized B-lymphocytes is due to a post-transcriptional block in expression of cytochrome b558.. Biochem J, 306 ( Pt 1), 141 - 145.
    • LEVIN M, NEWPORT MJ, DSOUZA S, KALABALIKIS P, BROWN IN, LENICKER HM, AGIUS PV, DAVIES EG, THRASHER A, KLEIN N, BLACKWELL JM (1995). FAMILIAL DISSEMINATED ATYPICAL MYCOBACTERIAL INFECTION IN CHILDHOOD - A HUMAN MYCOBACTERIAL SUSCEPTIBILITY GENE. LANCET, 345(8942), 79 - 83.
    • THRASHER AJ, DEALWIS M, CASIMIR CM, KINNON C, PAGE K, LEBKOWSKI J, SEGAL AW, LEVINSKY RJ (1995). FUNCTIONAL RECONSTITUTION OF THE NADPH-OXIDASE BY ADENOASSOCIATED VIRUS GENE-TRANSFER. BLOOD, 86(2), 761 - 765.

    1994

    • Thrasher AJ, Keep NH, Wientjes F, Segal AW (1994). Chronic granulomatous disease.. Biochim Biophys Acta, 1227(1-2), 1 - 24.

    1993

    • Thrasher A, Segal A, Casimir C (1993). Chronic granulomatous disease: towards gene therapy.. Immunodeficiency, 4(1-4), 327 - 333.

    1992

    • Thrasher A, Chetty M, Casimir C, Segal AW (1992). Restoration of superoxide generation to a chronic granulomatous disease-derived B-cell line by retrovirus mediated gene transfer.. Blood, 80(5), 1125 - 1129.
    • Abo A, Boyhan A, West I, Thrasher AJ, Segal AW (1992). Reconstitution of neutrophil NADPH oxidase activity in the cell-free system by four components: p67-phox, p47-phox, p21rac1, and cytochrome b-245.. J Biol Chem, 267(24), 16767 - 16770.

    • Thornhill SI, Schambach A, Howe SJ, Ulaganathan M, Grassman E, Williams D, Schiedlmeier B, Sebire NJ, Gaspar HB, Kinnon C, Baum C, Thrasher AJ (). Self-inactivating Gammaretroviral Vectors for Gene Therapy of X-linked Severe Combined Immunodeficiency.. Molecular Therapy, , - .