Prof Bobby Gaspar

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Personal Profile

Name: Bobby Gaspar Email: h.gaspar@ucl.ac.uk
Title: Prof Tel: 0044 207 905 2319
Department: ICH - Molecular Immunology Unit Fax: 0044 207 905 2810
Position: Professor of Paediatrics and Immunology Address: 30, Guilford Street, London , WC1N 1EH
Research Domain: Infection, Immunology & Inflammation, Personalised Medicine, Populations & Lifelong Health, Reproduction & Development Web Page:  

Profile

Research Description

Research in my group spans many aspects of primary immunodeficiencies including genetic diagnosis, molecular and cellular pathogenesis, haematopoietic stem cell transplantation and development of gene therapy. Current lab projects: 1) Understanding the molecular basis of TACI defects in Common Variable Immunodeficiency using in vitro and in vivo models 2) Development of T cell strategies to improve outcome following stem cell transplantation including genetic modification of T cells using suicide genes and generation of virus specific T cells 3) Development of gene therapy models for X-linked lymphoproliferative disease (XLP) and haemophagocytic lymphohistiocytosis (HLH) 4) Gene therapy for severe combined immunodeficiency (X-linked SCID and ADA SCID) + this includes implementation of clinical trials and development of new safer viral constructs Clinical projects 1) Outcome of stem cell transplantation for ADA deficiency 2) Understanding the systemic nature of ADA deficiency 3) Long term cognitive outcome in patients receiving stem cell transplants

Research Activities

Pathogenesis and management of primary immunodefic

T cell therapies

Education Description

UCL Collaborators

Prof Adrian Thrasher; Dr Waseem Qasim

External Collaborators

Publications

    2014

    • Buckland KF, Bobby Gaspar H (2014). Gene and cell therapy for children - New medicines, new challenges?. Adv Drug Deliv Rev, , - . doi:10.1016/j.addr.2014.02.010
    • Guengoer T, Teira P, Slatter M, Stuessi G, Stepensky P, Moshous D, Vermont C, Ahmad I, Shaw PJ, Telles da Cunha JM, Schlegel PG, Hough R, Fasth A, Kentouche K, Gruhn B, Fernandes JF, Lachance S, Bredius R, Resnick IB, Belohradsky BH, Gennery A, Fischer A, Gaspar HB, Schanz U, Seger R, Rentsch K, Veys P, Haddad E, Albert MH, Hassan M, Europe IEWP (2014). Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study. LANCET, 383(9915), 436 - 448. doi:10.1016/50140-6736(13)62069-3
    • 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
    • Al-Herz W, Bousfiha A, Casanova JL, Chatila T, Conley ME, Cunningham-Rundles C, Etzioni A, Franco JL, Gaspar HB, Holland SM, Klein C, Nonoyama S, Ochs HD, Oksenhendler E, Picard C, Puck JM, Sullivan K, Tang ML (2014). Primary immunodeficiency diseases: an update on the classification from the international union of immunological societies expert committee for primary immunodeficiency.. Front Immunol, 5, 162 - . doi:10.3389/fimmu.2014.00162
    • Gaspar HB (2014). Tearing RAGs apart.. Blood, 123(2), 156 - 157. doi:10.1182/blood-2013-11-535633
    • Buckland KF, Bobby Gaspar H (2014). Gene and cell therapy for children - New medicines, new challenges?. Advanced Drug Delivery Reviews, 73, 162 - 169. doi:10.1016/j.addr.2014.02.010
    • Gaspar HB, Hammarström L, Mahlaoui N, Borte M, Borte S (2014). The Case for Mandatory Newborn Screening for Severe Combined Immunodeficiency (SCID).. J Clin Immunol, , - . doi:10.1007/s10875-014-0029-0
    • 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
    • Passweg JR, Baldomero H, Peters C, Gaspar HB, Cesaro S, Dreger P, Duarte RF, Falkenburg JH, Farge-Bancel D, Gennery A, Halter J, Kröger N, Lanza F, Marsh J, Mohty M, Sureda A, Velardi A, Madrigal A (2014). Hematopoietic SCT in Europe: data and trends in 2012 with special consideration of pediatric transplantation.. Bone Marrow Transplant, , - . doi:10.1038/bmt.2014.55
    • Adams SP, Premachandra T, Wilson MC, Rashid S, Harvey K, Ifederu A, Gaspar HB (2014). Screening of Neonatal UK Dried Blood Spots Using a Duplex TREC Screening Assay. Journal of Clinical Immunology, , 1 - 8. doi:10.1007/s10875-014-0007-6

    2013

    • Qasim W, Gilmour K, Zhan H, Derniame S, McNicol AM, Ip W, Hiwarkar P, Veys P, Gaspar HB (2013). Interferon-γ capture T cell therapy for persistent Adenoviraemia following allogeneic haematopoietic stem cell transplantation.. Br J Haematol, 161(3), 449 - 452. doi:10.1111/bjh.12251
    • 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
    • Zhang L, Thrasher AJ, Gaspar HB (2013). Current progress on gene therapy for primary immunodeficiencies.. Gene Ther, , - . doi:10.1038/gt.2013.21
    • 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.
    • Passweg JR, Baldomero H, Bregni M, Cesaro S, Dreger P, Duarte RF, Falkenburg JHF, Kroeger N, Farge-Bancel D, Gaspar HB, Marsh J, Mohty M, Peters C, Sureda A, Velardi A, de Elvira CR, Madrigal A, Transpla EGBM (2013). Hematopoietic SCT in Europe: data and trends in 2011. BONE MARROW TRANSPLANTATION, 48(9), 1161 - 1167. doi:10.1038/bmt.2013.51
    • 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
    • Gaspar HB, Qasim W, Davies EG, Rao K, Amrolia PJ, Veys P (2013). How I treat severe combined immunodeficiency. Blood, , - . doi:10.1182/blood-2013-02-380105
    • 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
    • Güngör T, Teira P, Slatter M, Stussi G, Stepensky P, Moshous D, Vermont C, Ahmad I, Shaw PJ, da Cunha JM, Schlegel PG, Hough R, Fasth A, Kentouche K, Gruhn B, Fernandes JF, Lachance S, Bredius R, Resnick IB, Belohradsky BH, Gennery A, Fischer A, Gaspar HB, Schanz U, Seger R, Rentsch K, Veys P, Haddad E, Albert MH, Hassan M, on behalf of the Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation (2013). Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study.. Lancet, , - . doi:10.1016/S0140-6736(13)62069-3
    • Hiwarkar P, Gaspar HB, Gilmour K, Jagani M, Chiesa R, Bennett-Rees N, Breuer J, Rao K, Cale C, Goulden N, Davies G, Amrolia P, Veys P, Qasim W (2013). Impact of viral reactivations in the era of pre-emptive antiviral drug therapy following allogeneic haematopoietic SCT in paediatric recipients.. Bone Marrow Transplant, 48(6), 803 - 808. doi:10.1038/bmt.2012.221
    • 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
    • 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.

    2012

    • 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
    • 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.
    • 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
    • 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.
    • 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.
    • Hassan A, Booth C, Brightwell A, Allwood Z, Veys P, Rao K, Hönig M, Friedrich W, Gennery A, Slatter M, Bredius R, Finocchi A, Cancrini C, Aiuti A, Porta F, Lanfranchi A, Ridella M, Steward C, Filipovich A, Marsh R, Bordon V, Al-Muhsen S, Al-Mousa H, Alsum Z, Al-Dhekri H, Al Ghonaium A, Speckmann C, Fischer A, Mahlaoui N, Nichols KE, Grunebaum E, Al Zahrani D, Roifman CM, Boelens J, Davies EG, Cavazzana-Calvo M, Notarangelo L, Gaspar HB, Inborn Errors Working Party of the European Group for Blood and Marrow Transplantation and European Society for Immunodeficiency (2012). Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency.. Blood, 120(17), 3615 - 3624. doi:10.1182/blood-2011-12-396879
    • Booth C, Algar VE, Xu-Bayford J, Fairbanks L, Owens C, Gaspar HB (2012). Non-infectious lung disease in patients with adenosine deaminase deficient severe combined immunodeficiency.. J Clin Immunol, 32(3), 449 - 453. doi:10.1007/s10875-012-9658-3
    • Gaspar HB (2012). Gene therapy for ADA-SCID: defining the factors for successful outcome.. Blood, 120(18), 3628 - 3629. doi:10.1182/blood-2012-08-446559
    • Engelhardt KR, Gertz EM, Keles S, Schaeffer AA, Ceja R, Sassi A, Massaad MJ, Mellouli F, Benmustapha I, Khemiri M, Kilic SS, Etzioni A, Freeman AF, Thiel J, Schulze I, Al-Herz W, Metin A, Sanal O, Yeganeh M, Niehues T, Siepermann K, Unal E, Patiroglu T, Dasouki M, Yilmaz M, Genel F, Aytekin C, Kutukculer N, Somer A, Kilic M, Reisli I, Camcioglu Y, Gennery AR, Cant AJ, Jones A, Gaspar HB, Arkwright PD, Pietrogrande MC, Baz Z, Al-Tamemi S, Lougaris V, Lefranc G, Megarbane A, Boutros J, Galal N, Bejaoui M, Barbouche R, Geha RS, Chatila TA, Grimbacher B (2012). DOCK8 DEFICIENCY AND A DIAGNOSTIC SCORE TO DIFFERENTIATE IT FROM OTHER HYPER-IGE SYNDROMES.
    • 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.
    • 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

    2011

    • Booth C, Gilmour KC, Veys P, Gennery AR, Slatter MA, Chapel H, Heath PT, Steward CG, Smith O, O'Meara A, Kerrigan H, Mahlaoui N, Cavazzana-Calvo M, Fischer A, Moshous D, Blanche S, Pachlopnik Schmid J, Latour S, de Saint-Basile G, Albert M, Notheis G, Rieber N, Strahm B, Ritterbusch H, Lankester A, Hartwig NG, Meyts I, Plebani A, Soresina A, Finocchi A, Pignata C, Cirillo E, Bonanomi S, Peters C, Kalwak K, Pasic S, Sedlacek P, Jazbec J, Kanegane H, Nichols KE, Hanson IC, Kapoor N, Haddad E, Cowan M, Choo S, Smart J, Arkwright PD, Gaspar HB (2011). X-linked lymphoproliferative disease due to SAP/SH2D1A deficiency: a multicenter study on the manifestations, management and outcome of the disease.. Blood, 117(1), 53 - 62. doi:10.1182/blood-2010-06-284935
    • Booth C, Gaspar HB, Thrasher AJ (2011). Gene therapy for primary immunodeficiency.. Curr Opin Pediatr, 23(6), 659 - 666. doi:10.1097/MOP.0b013e32834cd67a
    • Palendira U, Low C, Chan A, Hislop AD, Ho E, Phan TG, Deenick E, Cook MC, Riminton DS, Choo S, Loh R, Alvaro F, Booth C, Gaspar HB, Moretta A, Khanna R, Rickinson AB, Tangye SG (2011). Molecular Pathogenesis of EBV Susceptibility in XLP as Revealed by Analysis of Female Carriers with Heterozygous Expression of SAP. PLOS BIOLOGY, 9(11), - . doi:10.1371/journal.pbio.1001187
    • 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
    • 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.
    • 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.
    • 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.
    • Zhang L, Montiel-Equihua CA, Thrasher AJ, Gaspar HB (2011). Dr Lin Zhang. HUMAN GENE THERAPY, 22(10), A53 - A54.
    • Skucek E, Butler S, Gaspar HB, Titman P (2011). Social outcome in children treated by haematopoietic cell transplant for congenital immunodeficiency. BONE MARROW TRANSPL, 46(10), 1314 - 1320. doi:10.1038/bmt.2010.318
    • Shaw SW, Bollini S, Nader KA, Gastadello A, Mehta V, Filppi E, Cananzi M, Gaspar HB, Qasim W, De Coppi P, David AL (2011). Autologous transplantation of amniotic fluid-derived mesenchymal stem cells into sheep fetuses.. Cell Transplant, 20(7), 1015 - 1031. doi:10.3727/096368910X543402
    • Brown L, Xu-Bayford J, Allwood Z, Slatter M, Cant A, Davies EG, Veys P, Gennery AR, Gaspar HB (2011). Neonatal Diagnosis of Severe Combined Immunodeficiency Leads to Significantly Improved Survival Outcome: The Case for Newborn Screening EDITORIAL COMMENT. OBSTET GYNECOL SURV, 66(7), 398 - 399. doi:10.1097/OGX.0b013e3182338432
    • 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
    • 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
    • Brown L, Xu-Bayford J, Allwood Z, Slatter M, Cant A, Davies EG, Veys P, Gennery AR, Gaspar HB (2011). Neonatal diagnosis of severe combined immunodeficiency leads to significantly improved survival outcome: the case for newborn screening. BLOOD, 117(11), 3243 - 3246. doi:10.1182/blood-2010-08-300384
    • 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
    • Gaspar HB (2011). Induced pluripotent stem cells and primary immunodeficiencies: a new frontier reached, a new world beyond?. J Allergy Clin Immunol, 127(6), 1408 - 1409. doi:10.1016/j.jaci.2011.02.038
    • Gholam C, Grigoriadou S, Gilmour KC, Gaspar HB (2011). Familial haemophagocytic lymphohistiocytosis: advances in the genetic basis, diagnosis and management.. Clin Exp Immunol, 163(3), 271 - 283. doi:10.1111/j.1365-2249.2010.04302.x

    2010

    • Samarasinghe S, Mancao C, Pule M, Nawroly N, Karlsson H, Brewin J, Openshaw P, Gaspar HB, Veys P, Amrolia PJ (2010). Functional characterization of alloreactive T cells identifies CD25 and CD71 as optimal targets for a clinically applicable allodepletion strategy. Blood, 115, 396 - 407.
    • 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.
    • 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.
    • Hislop AD, Palendira U, Leese AM, Arkwright PD, Rohrlich PS, Tangye SG, Gaspar HB, Lankester AC, Moretta A, Rickinson AB (2010). Impaired Epstein-Barr virus-specific CD8(+) T-cell function in X-linked lymphoproliferative disease is restricted to SLAM family-positive B-cell targets. BLOOD, 116(17), 3249 - 3257. doi:10.1182/blood-2009-09-238832
    • Shaw SWS, Bollini S, Nader KA, Gastadello A, Mehta V, Cananzi M, Gaspar HB, Qasim W, De Coppi P, David AL (2010). Wide spread and systemic engraftment after in utero autologous transplantation of amniotic fluid mesenchymal stem cells into sheep fetuses.
    • Gaspar HB (2010). Bone Marrow Transplantation and Alternatives for Adenosine Deaminase Deficiency. IMMUNOL ALLERGY CLIN, 30(2), 221 - +. doi:10.1016/j.iac.2010.01.002
    • Shaw SWS, Bollini S, Nader KA, Gastadello A, Mehta V, Filppi E, Cananzi M, Gaspar HB, Qasim W, De Coppi P, David AL (2010). Widespread Engraftment after In Utero Autologous Transplantation of Amniotic Fluid Derived Mesenchymal Stem Cells into Sheep Fetuses..
    • Walshe D, Gaspar HB, Thrasher AJ, Cale CM, Gilmour KC (2010). STAT5 tyrosine phosphorylation for diagnosis and monitoring of patients with severe combined immunodeficiency.
    • 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.

    2009

    • Gaspar HB, Aiuti A, Porta F, Candotti F, Hershfield MS, Notarangelo LD (2009). How I treat ADA deficiency. Blood, , - .
    • Qasim W, Gaspar HB, Thrasher AJ (2009). Progress and prospects: gene therapy for inherited immunodeficiencies. Gene Ther., , - .
    • Straathof KC, Rao K, Eyrich M, Hale G, Bird P, Berrie E, Brown L, Schlegel PG, Goulden N, Gaspar B, Gennery A, Landais P, Davies EG, Brenner MK, Veys P, Amrolia P (2009). A novel antibody-based minimal-intensity conditioning regimen for children with severe organ toxicity or DNA repair disorders.
    • 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.
    • 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.
    • Ljungman P, Bregni M, Brune M, Cornelissen J, Witte TD, Dini G, Einsele H, Gaspar HB, Gratwohl A, Passweg J, Peters C, Rocha V, Saccardi R, Schouten H, Sureda A, Tichelli A, Velardi A, Niederwieser D (2009). Allogeneic and autologous transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe 2009. Bone Marrow Transplant., , - .
    • 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.
    • 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.
    • Straathof KC, Rao K, Eyrich M, Hale G, Bird P, Berrie E, Brown L, Adams S, Schlegel PG, Goulden N, Gaspar HB, Gennery AR, Landais P, Davies EG, Brenner MK, Veys PA, Amrolia PJ (2009). Haemopoietic stem-cell transplantation with antibody-based minimal-intensity conditioning: a phase 1/2 study. Lancet, 374(9693), 912 - 920.
    • 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.
    • Griffith LM, Cowan MJ, Notarangelo LD, Puck JM, Buckley RH, Candotti F, Conley ME, Fleisher TA, Gaspar HB, Kohn DB, Ochs HD, O'Reilly RJ, Rizzo JD, Roifman CM, Small TN, Shearer WT (2009). Improving cellular therapy for primary immune deficiency diseases: Recognition, diagnosis, and management. Journal of Allergy and Clinical Immunology, 124(6), 1152 - 1160.
    • 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, 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.
    • Booth C, Gaspar HB (2009). Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID). Biologics., 3, 349 - 358.

    2008

    • Thapar N, Lindley KJ, Kiparissi F, Elawad MA, Ashworth M, Veys P, Gaspar HB, Hill SM, Milla PJ, Shah N (2008). Treatment of intractable ulcerating enterocolitis of infancy by allogeneic bone marrow transplantation.. Clin Gastroenterol Hepatol, 6(2), 248 - 250. doi:10.1016/j.cgh.2007.11.019
    • 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
    • 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, , - .
    • 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.
    • 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.
    • 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.
    • Bennour E, Ferrand C, Remy-Martin JP, Certoux JM, Gorke S, Qasim W, Gaspar HB, Baumert T, Duperrier A, Deschamps M, Fehse B, Tiberghien P, Robinet E (2008). Abnormal expression of only the CD34 part of a transgenic CD34/herpes simplex virus-thymidine kinase fusion protein is associated with ganciclovir resistance. Hum.Gene Ther., 19(7), 699 - 709.
    • 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.
    • 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 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.
    • 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.

    2007

    • 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
    • Booth C, Hershfield M, Notarangelo L, Buckley R, Hoenig M, Mahlaoui N, Cavazzana-Calvo M, Aiuti A, Gaspar HB (2007). Management options for adenosine deaminase deficiency; proceedings of the EBMT satellite workshop (Hamburg, March 2006).. Clin Immunol, 123(2), 139 - 147. doi:10.1016/j.clim.2006.12.009
    • 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.
    • 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.
    • 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
    • Booth C, Hershfield M, Notarangelo L, Buckley R, Hoenig M, Mahlaoui N, Cavazzana-Calvo M, Aiuti A, Gaspar HB (2007). Management options for adenosine deaminase deficiency; proceedings of the EBMT satellite workshop (Hamburg, March 2006). CLIN IMMUNOL, 123(2), 139 - 147. doi:10.1016/j.clin.2006.12.009
    • 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.
    • Cohen JM, Sebire NJ, Harvey J, Gaspar HB, Cathy C, Jones A, Rao K, Cubitt D, Amrolia PJ, Davies EG, Veys P (2007). Successful treatment of lymphoproliferative disease complicating primary immunodeficiency/immunodysregulatory disorders with reduced-intensity allogeneic stem-cell transplantation.. Blood, 110(6), 2209 - 2214. doi:10.1182/blood-2006-12-062174
    • Pan-Hammarstrom Q, Salzer U, Du L, Bjorkander J, Cunningham-Rundles C, Nelson DL, Bacchelli C, Gaspar HB, Offer S, Behrens TW, Grimbacher B, Hammarstrom L (2007). Reexamining the role of TACI coding variants in common variable immunodeficiency and selective IgA deficiency. Nature Genetics, 39(4), 429 - 430.
    • 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
    • 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, 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

    2006

    • Chatziandreou I, Gilmour KC, McNicol AM, Costabile M, Sinclair J, Cubitt D, Campbell JD, Kinnon C, Qasim W, Bobby GH (2006). Capture and generation of adenovirus specific T cells for adoptive immunotherapy. Br J Haematol, , - .
    • Worth A, Thrasher AJ, Gaspar HB (2006). Autoimmune lymphoproliferative syndrome: molecular basis of disease and clinical phenotype. Br J Haematol, 133(2), 124 - 140.
    • Weber MA, Malone M, Thrasher A, Gaspar HB, Sebire NJ (2006). Cutaneous graft versus host disease-like histopathological features following gene therapy.
    • 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.
    • 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.
    • Cohen JM, Rogers V, Gaspar HB, Jones A, Davies EG, Rao K, McCloskey DJ, Gilmour K, Wynn R, Amrolia PJ, Veys P (2006). Serial transplantation of mismatched donor hematopoietic cells between HLA-identical sibling pairs with congenital immunodeficiency: in vivo tolerance permits rapid immune reconstitution following T-replete transplantation without GVHD in the secondary recipient. Blood, 108(6), 2124 - 2126.
    • 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.
    • 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.
    • Amrolia PJ, Muccioli-Casadei G, Huls H, Adams S, Durett A, Gee A, Yvon E, Weiss H, Cobbold M, Gaspar HB, Rooney C, Kuehnle I, Ghetie V, Schindler J, Krance R, Heslop HE, Veys P, Vitetta E, Brenner MK (2006). Adoptive immunotherapy with allodepleted donor T-cells improves immune reconstitution after haploidentical stem cell transplantation. Blood, 108(6), 1797 - 1808. doi:10.1182/blood-2006-02-001909
    • 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.
    • Sanchez JJ, Monaghan G, Borsting C, Norbury G, Morling N, Gaspar HB (2006). Carrier Frequency of a Nonsense Mutation in the Adenosine Deaminase (ADA) Gene Implies a High Incidence of ADA-deficient Severe Combined Immunodeficiency (SCID) in Somalia and a Single, Common Haplotype Indicates Common Ancestry. Ann.Hum.Genet., , - .
    • 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.

    2005

    • 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.
    • 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
    • Gaspar HB, Thrasher AJ (2005). Gene therapy for severe combined immunodeficiencies. EXPERT OPIN BIOL THER, 5(9), 1175 - 1182.
    • 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.
    • Plunkett FJ, Franzese O, Belaramani LL, Fletcher JM, Gilmour KC, Sharifi R, Khan N, Hislop AD, Cara A, Salmon M, Gaspar HB, Rustin MH, Webster D, Akbar AN (2005). The impact of telomere erosion on memory CD8(+) T cells in patients with X-linked lymphoproliferative syndrome. MECH AGEING DEV, 126(8), 855 - 865.
    • Williams AP, Hourihane JOB, Hodges E, Gaspar HB, Smith J (2005). Multisystem disease in CAEBV infection. Clinical Immunology, 115, S248 - S248.
    • Kampmann B, Cubitt D, Walls T, Naik P, Depala M, Samarasinghe S, Robson D, Hassan A, Rao K, Gaspar H, Davies G, Jones A, Cale C, Gilmour K, Real M, Foo M, Bennett-Rees N, Hewitt A, Amrolia P, Veys P (2005). Improved outcome for children with disseminated adenoviral infection following allogeneic stem cell transplantation. British Journal of Haematology, 130(4), 595 - 603.
    • Rao K, Amrolia PJ, Jones A, Cale CM, Naik P, King D, Davies GE, Gaspar HB, Veys PA (2005). Improved survival after unrelated donor bone marrow transplant in children with primary immunodeficiency using a reduced intensity conditioning regimen. Blood, 105(2), 879 - 885. doi:10.1182/blood-2004-03-0960
    • 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.
    • Thrasher AJ, Gaspar HB (2005). Gene therapy for lympho-hematopoietic disorders. Current Hematology Reports, 4(4), 305 - 309.
    • 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.
    • 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.
    • Lankester AC, Visser LF, Hartwig NG, Bredius RG, Gaspar HB, van der BM, van Tol MJ, Gross TG, Egeler RM (2005). Allogeneic stem cell transplantation in X-linked lymphoproliferative disease: two cases in one family and review of the literature. BONE MARROW TRANSPLANT, 36(2), 99 - 105.
    • 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.
    • Cohen J , Gandhi M , Naik P , Cubitt D , Rao K , Thaker U , Davies EG , Gaspar HB , Amrolia P , Veys P (2005). Increased incidence of EBV-related disease following paediatric stem cell transplantation with reduced-intensity conditioning.. Br J Haematol, 129, 229 - 239.

    2004

    • Eastwood D, Gilmour KC, Nistala K, Meaney C, Chapel H, Sherrell Z, Webster AD, Davies EG, Jones A, Gaspar HB (2004). Prevalence of SAP gene defects in male patients diagnosed with common variable immunodeficiency. Clin.Exp.Immunol., 137(3), 584 - 588.
    • Qasim W, Gaspar HB, Thrasher AJ (2004). Gene therapy for severe combined immune deficiency. EXPERT REV MOL MED, 2004, 1 - 15.
    • Kampmann B, Cubitt D, Naik P, Depala M, Hassan A, Rao K, Gaspar H, Davies G, Jones A, Cale C, Gilmour K, Amrolia P, Real M, Foo M, Bennet-Rees N, Hewitt N, Veys P (2004). Which children die from overwhelming adenoviral infection following haematopoietic stem cell transplantation?.
    • Gilmour K , Eastwood D , Nistala C , Meaney C , Chapel H , Sherrell Z , Webster DA , Davies EG , Jones A , Gaspar HB (2004). Prevalence of SAP gene defects in male patients diagnosed with common variable immunodeficiency.
    • 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.
    • Adams S, Veys P, Hollifield J, Gaspar B (2004). Cell lineage specific chimerism in post-haematopoietic stem cell transplant patients. Bone Marrow Transplantation, 33, S101 - S102.
    • 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.
    • 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.
    • Sebire NJ, Malone M, Shah N, Anderson G, Gaspar HB, Cubitt WD (2004). Pathology of astrovirus associated diarrhoea in a paediatric bone marrow transplant recipient. Journal of Clinical Pathology, 57(9), 1001 - 1003.
    • 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
    • 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
    • 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.
    • Albuquerque W, Gaspar HB (2004). Bilateral sensorineural deafness in adenosine deaminase-deficient severe combined immunodeficiency. The Journal of Pediatrics, 144(2), 278 - 280.
    • Eastwood D, Gilmour KC, Gaspar HB (2004). Molecular diagnosis of congenital immunodeficiency. Methods in Molecular Medicine, 91, 91 - 108.
    • 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.
    • Sebire NJ, Malone M, Anderson G, Shah N, Gaspar HB, Cubitt WD (2004). Human intestinal astrovirus infection in an immunocomprimised child following bone marrow transplantation.
    • Sebire NJ, Malone M, Anderson G, Shah N, Gaspar HB, Cubitt WD (2004). "Human intestinal astrovirus infection in an immunocompromised child following bone marrow transplantation".
    • 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

    2003

    • 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
    • Gaspar HB, Howe S, Thrasher AJ (2003). Gene therapy progress and prospects: gene therapy for severe combined immunodeficiency. Gene Therapy, 10(24), 1999 - 2004.
    • Gilmour K, Gaspar HB (2003). Pathogenesis and diagnosis of X-linked lymphoproliferative disease. Expert Review of Molecular Diagnostics, 3(5), 549 - 561.

    2002

    • 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
    • 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
    • Kinnon C, Gaspar HB (2002). Genetics of Inherited Immunodeficiency Diseases and Diagnostic Techniques. In (Ed.), Manual of Clinical Laboratory Immunology (pp. 810 - 812). : ASM Press.
    • 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.
    • 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
    • Gaspar HB , Amrolia P , Hassan A , Webb D , Jones A , Sturt N , Vergani G , Pagliuca A , Mufti G , Hadzic N , Davies G , Veys P (2002). Non-myeloablative stem cell transplantation for congenital immunodeficiencies. Recent Results Cancer Research, , 134 - 142.

    2001

    • Rogers MH, Lwin R, Fairbanks L, Gerritsen B, Gaspar HB (2001). Cognitive and behavioral abnormalities in adenosine deaminase deficient severe combined immunodeficiency. The Journal of Pediatrics, 139(1), 44 - 50.
    • Gaspar HB, Kinnon C (2001). X-linked agammaglobulinemia. IMMUNOL ALLERGY CLIN, 21(1), 23 - +.
    • (2001). Cognitive and behavioural abnormalities in adenosine deaminase deficient severe combined immunodeficiency. BONE MARROW TRANSPLANATATION, 27, 256 - .
    • Gaspar HB, Gilmour KC, Jones AM (2001). Severe combined immunodeficiency - molecular pathogenesis and diagnosis. Archives of Disease in Childhood, 84(2), 169 - 173.
    • Gilmour K , Fujii H CT, Davies EG, Kinnon C , Gaspar HB (2001). Defective expression of the interleukin-2/interleukin-15 receptor beta subunit leads to a natural killer cell - deficient form of severe combined immunoodefiency. Blood, 98(3), 877 - 879.
    • 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.
    • Nistala K , Gilmour KC , Cranston T , Davies EG , Goldblatt D , Gaspar HB , Jones AM (2001). X-linked lymphoproliferative disease: three atypical cases.. Clin.Exp.Immunol., 126, 126 - 130.
    • Gilmour KC, Cranston T, Loughlin S, Gwyther J, Lester T, Espanol T, Hernandez M, Savoldi G, Davies EG, Abinun M, Kinnon C, Jones A, Gaspar HB (2001). Rapid protein-based assays for the diagnosis of T-B+ severe combined immunodeficiency. Br.J Haematol., 112(3), 671 - 676.
    • (2001). Screening for genetic defects in primary immunodeficiencies. CPD Bulletin Immunology and Allergy, 2, 3 - 7.
    • Nistala K, Gilmour KC, Cranston T, Davies EG, Goldblatt D, Gaspar HB, Jones AM (2001). X-linked lymphoproliferative disease: three atypical cases.. Clin Exp Immunol, 126(1), 126 - 130.

    2000

    • Ramanan AV, Hussain K, Hird M, Gaspar HB (2000). Short limbed skeletal dysplasia associated with combined immunodeficiency and congenital subglottic stenosis: a new constellation of features. Clinical Dysmorphology, 9(3), 173 - 176.
    • Cranston T, Gilmour K, Loughlin S, Walshe D, Gaspar HB, Lester T, Heath S, Jones AM (2000). Strategy for molecular diagnosis of immunodeficiency at Great Ormond Street Hospital: a multidisciplinary approach. Journal of Medical Genetics, 37, 624 - .
    • 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.
    • Amrolia P, Gaspar HB, Hassan A, Webb D, Jones A, Sturt N, Mieli-Vergani G, Pagliuca A, Mufti G, Hadzic N, Davies G, Veys P (2000). Nonmyeloablative stem cell transplantation for congenital immunodeficiencies. Blood, 96(4), 1239 - 1246.
    • Gaspar HB, Ferrando M, Caragol I, Hernandez M, Bertran JM, De gracia X, Lester T, Kinnon C, Ashton E, Espanol T (2000). Kinase mutant Btk results in atypical X-linked agammaglobulinaemia phenotype. Clinical and Experimental Immunology, 120(2), 346 - 350.
    • 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.
    • Jones AM, Gaspar HB (2000). Immunogenetics: changing the faces of immunodeficiency. Journal of Clinical Pathology, 53(1), 60 - 65.
    • Kinnon C, Gaspar HB (2000). Genetics of inherited immunodeficiency diseases and diagnostic techniques. In (Ed.), Manual for Clinical Laboratory Immunology (pp. - ). : .
    • Gaspar HB, Conley ME (2000). Early B cell defects. Clinical and Experimental Immunology, 119(3), 383 - 389.

    1999

    • 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 - .
    • 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.

    1998

    • Gaspar HB, Lester T, Levinsky RJ, Kinnon C (1998). Bruton's tyrosine kinase expression and activity in X-linked agammaglobulinaemia (XLA): the use of protein analysis as a diagnostic indicator of XLA.. Clin Exp Immunol, 111(2), 334 - 338.
    • Gaspar HB, Lester T, Levinsky RJ, Kinnon C (1998). Analysis of Bruton's tyrosine kinase protein expression and activity in patients with X-linked agammaglobulinemia (XLA):the use of protein analysis as a diagnostic indicator of XLA. Clinical and Experimental Immunology, 111(2), 334 - 338.
    • MacCarthy-Morrogh L, Gaspar HB, Wang YC, Katz F, Thompson L, Layton M, Jones AM, Kinnon C (1998). Absence of expression of the Wiskott-Aldrich syndrome protein in peripheral blood cells of Wiskott-Aldrich syndrome patients.. Clin Immunol Immunopathol, 88(1), 22 - 27.

    1997

    • Gaspar HB, Goldblatt D (1997). Immunodeficiency syndromes and recurrent infection. Br.J.Hosp.Med., 58(11), 565 - 568.
    • Gaspar HB, Goldblatt D (1997). Investigation of the immune system in recurrent infection. Br.J.Hosp.Med., 58(10), 517 - 520.

    1995

    • Gaspar HB, Bradley LA, Katz F, Lovering RC, Roifman CM, Morgan G, Levinsky RJ, Kinnon C (1995). Mutation analysis in Bruton's tyrosine kinase, the X-linked agammaglobulinaemia gene, including identification of an insertional hotspot.. Hum Mol Genet, 4(4), 755 - 757.

    1994

    • Genevier HC, Hinshelwood S, Gaspar HB, Rigley KP, Brown D, Saeland S, Rousset F, Levinsky RJ, Callard RE, Kinnon C (1994). Expression of Bruton's tyrosine kinase protein within the B cell lineage.. Eur J Immunol, 24(12), 3100 - 3105. doi:10.1002/eji.1830241228

    • 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, , - .