Prof Amit Nathwani

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Personal Profile

Name: Amit Nathwani Email: amit.nathwani@ucl.ac.uk
Title: Prof Tel: 2076796225
Department: Research Department of Haematology Fax: 2076796222
Position: Address: UCL Cancer Institute, Paul O'Gorman Building, 72 Huntley Street, London, WC1E 6BT
Research Domain: Basic Life Sciences, Cancer, Experimental Medicine, Personalised Medicine Web Page:  

Profile

Research Description

The main thrust of my research is the development of novel treatment avenues for haematological disorders and cancer using gene transfer technology. I am in addition interested in stem cell biology
My group has developed a distinctive approach for gene therapy of haemophilia B using a self complementary adeno-associated viral vector (AAV), in collaboration with St Jude Children's Research Hospital in Memphis Tennessee. This vector is significantly more potent than conventional AAV vectors. We have demonstrated the preclinical safety and efficacy of this new vector system and are about to commence a Phase I/II study in subjects with severe haemophilia B. Other clinical targets that are currently being evaluated include: Haemophilia A, Congenital FVII and FX deficiency, urea cycle and lysosomal storage disorders.
Haemato-oncology research is focused on the development of two emerging strategies: immunotherapy and anti-angiogenesis. Gene-therapy approaches are central to each of these strategies. Immunotherapy strategies using engineered T cells are being evaluated for the treatment of chronic lymphocytic leukaemia, a disease that remains incurable. Angiogenesis is required for the growth of tumours including haematological malignancies thus making it an important target for anti-cancer strategies. Long-term expression of an angiogenesis inhibitor is likely to be required and the role of gene therapy-mediated delivery of these agents is being evaluated.
Recently we have begun a program of research on the use of induced pluripotent stem (iPS) cells for the treatment of degenerative conditions as well as disease models. Through funding from the UKRMP/MRC we evaluating methods for the generation of iPS cells in a GMP environment. In addition, through EU framework support we are assessing the potential of iPS cells as a source of cells for the treatment of malignancies and or organ failure.  
Gene therapy for haematological malignancies: My haemato-oncology research is focused on the development of two emerging strategies: immunotherapy and anti-angiogenesis. Gene-therapy approaches are central to each of these strategies.

Immunotherapy strategies are directed against chronic lymphocytic leukaemia, a disease that remains incurable. Current emphasis is on the adoptive transfer of bi-specific gene modified T cells which recognise CMV infected cells through their native receptor and CLL via a tumour antigen specific receptor. Our ultimate goal is to adoptively transfer bi-specific cells immediately after front line therapy in patients with poor prognosis CLL.
Angiogenesis is required for the growth of tumours including haematological malignancies thus making it an important target for anti-cancer strategies. Long-term expression of an angiogenesis inhibitor is likely to be required for the successful treatment of malignancies. Therefore, gene therapy-mediated delivery of these agents is an alternative way to provide long-term expression of these therapeutic proteins. Vectors expressing a number of different angiogenesis inhibitors are currently being designed and tested in vitro and in vivo in murine models of leukaemia and lymphoma.


Research Activities

Application of liver cell biology to developing ne

Clinical, genetic and cell biology studies of hereditary movement disorders: dystonia, Huntington’s disease, Hereditary spastic paraplegia

Gene Therapy for Cancer

Gene therapy for haematological disorders

Gene transfer and regenerative medicine

Gene- and oligonucleotide-based therapies

Inhibition of angiogenesis

stem cell biology

Education Description

A. Medical students
i) Introduction to Blood groups
ii) Basic blood transfusion
iii) Introduction to leukaemias including CLL

B. Foundation year 1 and 2 teaching
Use of blood products, decision pathway

C. SpR Teaching at the NBS and UCLH
(i) Basic blood transfusion
(ii) Use of blood products and special requirements
(iii) Red cell transfusion, reactions and substitutes
(iv) Management of transfusion reaction
(v) Treatment options in CLL

D. MSc course in transfusion
Clinical decision in the use of blood products

E. UCLH Trust Induction Course in Transfusion
Blood transfusion training for HO and SHO

UCL Collaborators

Dr Aris Tagalakis; Prof Adrian Thrasher; Prof John Hardy; Dr Bernard Khoo; Prof Jim Owen; Prof Barry Fuller; Prof Alethea Tabor; Prof Peter Hindmarsh; Dr Clare Selden; Dr Paul Simons; Prof Dimitri Michael Kullmann; Prof Edward Tuddenham; Prof Stephen Moss; Prof Tom Warner

External Collaborators

Publications

    2012

    • Rahim AA, Wong AM, Ahmadi S, Hoefer K, Buckley SM, Hughes DA, Nathwani AN, Baker AH, McVey JH, Cooper JD, Waddington SN (2012). In utero administration of Ad5 and AAV pseudotypes to the fetal brain leads to efficient, widespread and long-term gene expression.. Gene Ther, 19(9), 936 - 946. doi:10.1038/gt.2011.157
    • Mattar CN, Nathwani A, Rosales C, Waddington S, Johana N, Ng X-W, Biswas A, Choolani M, Chan JKY (2012). Early AAV-Mediated Intrauterine Gene Therapy at 0.4G in Non-Human Primates.
    • Sack BK, Merchant S, Markusic DM, Byrne BJ, Herzog RW, Nathwani AC, Davidoff AM (2012). Transient B cell depletion or improved transgene expression by codon optimization promote tolerance to factor VIII in gene therapy. PLoS ONE, 7(5), - . doi:10.1371/journal.pone.0037671
    • McIntosh JH, Cochrane M, Cobbold S, Waldmann H, Nathwani SA, Davidoff AM, Nathwani AC (2012). Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine.. Gene Ther, 19(1), 78 - 85. doi:10.1038/gt.2011.64
    • Della Peruta M, Binny C, Nathwani D, McIntosh J, Rosales C, Davidoff AM, Williams R, Chokshi S, Nathwani AC (2012). DEVELOPMENT OF A LIVER-SPECIFIC, TUMOUR-SELECTIVE RECOMBINANT ADENO-ASSOCIATED VIRUS VECTOR TO TARGET HEPATOCELLULAR CARCINOMA.
    • Theodoulou A, Berryman J, Nathwani A, Scully M (2012). Comparison of cryoprecipitate with fibrinogen concentrate for acquired hypofibrinogenaemia. Transfusion and Apheresis Science, 46(2), 159 - 162. doi:10.1016/j.transci.2011.11.005
    • McIntosh JH, Cochrane M, Nathwani SA, Nathwani AC, Cobbold S, Waldmann H, Davidoff AM (2012). Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine. Gene Therapy, 19(1), 78 - 85. doi:10.1038/gt.2011.64
    • Theodoulou A, Berryman J, Nathwani A, Scully M (2012). Comparison of cryoprecipitate with fibrinogen concentrate for acquired hypofibrinogenaemia.. Transfus Apher Sci, 46(2), 159 - 162. doi:10.1016/j.transci.2011.11.005

    2011

    • Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC, Chowdary P, Riddell A, Pie AJ, Harrington C, O'Beirne J, Smith K, Pasi J, Glader B, Rustagi P, Ng CY, Kay MA, Zhou J, Spence Y, Morton CL, Allay J, Coleman J, Sleep S, Cunningham JM, Srivastava D, Basner-Tschakarjan E, Mingozzi F, High KA, Gray JT, Reiss UM, Nienhuis AW, Davidoff AM (2011). Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.. N Engl J Med, 365(25), 2357 - 2365. doi:10.1056/NEJMoa1108046
    • David AL, McIntosh J, Peebles DM, Cook T, Waddington S, Weisz B, Wigley V, Abi-Nader K, Boyd M, Davidoff AM, Nathwani AC (2011). Recombinant adeno-associated virus-mediated in utero gene transfer gives therapeutic transgene expression in the sheep.. Hum Gene Ther, 22(4), 419 - 426. doi:10.1089/hum.2010.007
    • Casper CJ, Warner TT, Taanman JW, Nathwani AC (2011). Generation of iPS-derived dopaminergic neurons as an in-vitro model of Rapid-Onset Dystonia Parkinsonism. HUMAN GENE THERAPY, 22(10), A42 - A43.
    • 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.
    • 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, , - .
    • Rahim AA, Wong AM, Ahmadi S, Hoefer K, Buckley SM, Hughes DA, Nathwani AN, Baker AH, McVey JH, Cooper JD, Waddington SN (2011). In utero administration of Ad5 and AAV pseudotypes to the fetal brain leads to efficient, widespread and long-term gene expression. Gene Ther., , - . doi:10.1038/gt.2011.157
    • Herzog RW, Davidoff AM, Markusic DM, Nathwani AC (2011). AAV Vector Biology in Primates: Finding the Missing Link?. Mol.Ther., 19(11), 1923 - 1924. doi:10.1038/mt.2011.218
    • Raj D, Davidoff AM, Nathwani AC (2011). Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challenges.. Expert Rev Hematol, 4(5), 539 - 549. doi:10.1586/ehm.11.48
    • Fagone P, Wright JF, Nathwani AC, Nienhuis AW, Davidoff AM, Gray JT (2011). Systemic Errors in Quantitative Polymerase Chain Reaction Titration of Self-Complementary Adeno-Associated Viral Vectors and Improved Alternative Methods. Hum.Gene Ther., , - . doi:10.1089/hum.2011.104
    • McIntosh JH, Cochrane M, Cobbold S, Waldmann H, Nathwani SA, Davidoff AM, Nathwani AC (2011). Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine. Gene Ther., , - . doi:10.1038/gt.2011.64
    • Nathwani AC, Tuddenham EGD, Rosales C, Macintosh J, Chowdary P, Riddell A, Aghighi S, Lilley P, Yee T, Griffioen A, Pie J, Harrington C, Glader B, Ng CYC, Kaye M, Zhou JF, Allay J, Coleman J, Sleep S, High K, Mingozzi F, Gray JT, Reiss UM, Nienhuis AW, Davidoff AM (2011). A phase i/ii clinical trial entailing peripheral vein administration of a novel self complementary adeno-associated viral vector encoding human fix for haemophilia b gene therapy.
    • Hillmen P, Gribben JG, Follows GA, Milligan D, Sayala HA, Moreton P, Oscier DG, Dearden CE, Kennedy DB, Pettitt AR, Nathwani A, Cohen D, Rawstron A, Pocock CF (2011). RITUXIMAB PLUS CHLORAMBUCIL (R-CHLORAMBUCIL) AS FIRST-LINE TREATMENT FOR CHRONIC LYMPHOCYTIC LEUKAEMIA (CLL): FINAL ANALYSIS OF AN OPEN-LABEL PHASE II STUDY.
    • 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
    • Nathwani A, Tuddenham EGD, Rosales C, Macintosh J, Chowdary P, Riddell A, Aghighi S, Griffioen A, Pie J, Harrington C, Glader B, Ng CYC, Kay M, Zhou J, Allay J, Coleman J, Sleep S, High K, Mingozzi F, Gray JT, Reiss UM, Nienhuis AW, Davidoff AM (2011). A Phase I/II clinical trial entailing peripheral vein administration of a novel self complementary adeno-associated viral vector encoding human FIX for Haemophilia B gene therapy. HUMAN GENE THERAPY, 22(10), A20 - A20.
    • Porakishvili N, Memon A, Vispute K, Kulikova N, Clark EA, Rai KR, Nathwani A, Damle RN, Chiorazzi N, Lydyard PM (2011). CD180 functions in activation, survival and cycling of B chronic lymphocytic leukaemia cells.. Br J Haematol, 153(4), 486 - 498. doi:10.1111/j.1365-2141.2011.08605.x
    • Farren TW, Liu F, Macey MG, Jenner M, Prentice A, Nathwani A, Agrawal SG (2011). CD160: a novel approach to minimal residual disease (MRD) assessment in chronic lymphocytic leukaemia (CLL).
    • Hillmen P, Gribben JG, Follows GA, Milligan D, Sayala HA, Moreton P, Oscier DG, Dearden CE, Kennedy DB, Pettitt AR, Nathwani A, Cohen D, Rawstron A, Pocock CFE, Sub-Grp NCRICLL (2011). Final response assessment of the CLL208 trial: an open-label phase II study to investigate the safety and efficacy of rituximab plus chlorambucil in previously untreated patients with CD20-positive B-cell chronic lymphocytic leukaemia (CLL)..
    • Raj D, Peggs K, Pule M, Linch D, Davies M, Nathwani A (2011). Adoptive immunotherapy for patients with chronic lymphocytic leukaemia using a chimeric antigen receptor targeting CD23.
    • Della Peruta M, Binny C, Nathwani D, Chokshi S, McIntosh J, Rosales C, Davidoff AM, Williams R, Nathwani AC (2011). The development of a tumour-selective recombinant adeno-associated virus vector to target hepatocellular carcinoma. HUMAN GENE THERAPY, 22(10), A113 - A113.
    • McIntosh J, Lenting P, Tuddenham E, Nawathe S, Waddington S, Gray J, Davidoff A, Nathwani A (2011). AAV mediated Factor VIII expression from a novel expression cassette with a potent codon-optimised FVIII construct. HUMAN GENE THERAPY, 22(10), A28 - A28.
    • Mattar CN, Nathwani AC, Waddington SN, Dighe N, Kaeppel C, Nowrouzi A, McIntosh J, Johana NB, Ogden B, Fisk NM, Davidoff AM, David A, Peebles D, Valentine MB, Appelt JU, von KC, Schmidt M, Biswas A, Choolani M, Chan JK (2011). Stable Human FIX Expression After 0.9G Intrauterine Gene Transfer of Self-complementary Adeno-associated Viral Vector 5 and 8 in Macaques. Mol.Ther., 19(11), 1950 - 1960. doi:10.1038/mt.2011.107
    • Denbo JW, Williams RF, Orr WS, Sims TL, Ng CY, Zhou J, Spence Y, Morton CL, Nathwani AC, Duntsch C, Pfeffer LM, Davidoff AM (2011). Continuous local delivery of interferon-beta stabilizes tumor vasculature in an orthotopic glioblastoma xenograft resection model. Surgery, 150(3), 497 - 504. doi:10.1016/j.surg.2011.07.044
    • Allay JA, Sleep S, Long S, Tillman DM, Clark R, Carney G, Fagone P, McIntosh JH, Nienhuis AW, Davidoff AM, Nathwani AC, Gray JT (2011). Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial. HUM GENE THER, 22(5), 595 - 604. doi:10.1089/hum.2010.202
    • Nathwani AC, Rosales C, McIntosh J, Rastegarlari G, Nathwani D, Raj D, Nawathe S, Waddington SN, Bronson R, Jackson S, Donahue RE, High KA, Mingozzi F, Ng CY, Zhou J, Spence Y, McCarville MB, Valentine M, Allay J, Coleman J, Sleep S, Gray JT, Nienhuis AW, Davidoff AM (2011). Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins. Mol.Ther., 19(5), 876 - 885. doi:10.1038/mt.2010.274
    • Rosales C, McIntosh J, Hanley J, Nathwani A, Morton C, Davidoff A (2011). A novel epigenetic modifier significantly improves recombinant adeno-associated virus vector mediated transgene expression in mice. HUMAN GENE THERAPY, 22(10), A107 - A107.

    2010

    • Tracey L, Streck CJ, Williams RF, Davidoff AM, Du Z, Pfeffer LM, Nathwani AC (2010). NF-κB activation mediates resistance to IFNΒ in MLL-rearranged acute lymphoblastic leukemia. Leukemia, 24(4), 806 - 812. doi:10.1038/leu.2010.2
    • Sims TL, McGee M, Williams RF, Myers AL, Tracey L, Hamner JB, Ng C, Wu J, Gaber MW, McCarville B, Nathwani AC, Davidoff AM (2010). IFN-beta restricts tumor growth and sensitizes alveolar rhabdomyosarcoma to ionizing radiation. Mol.Cancer Ther., 9(3), 761 - 771. doi:10.1158/1535-7163.MCT-09-0800
    • Porakishvili N, Lydyard P, Bremser A, Vispute K, Memon A, Kulikova N, Clarke E, Rai KR, Nathwani A, Damle RN, Chiorazzi N (2010). B Cell Chronic Lymphocytic Leukemia (B CLL) Cells Unresponsive to CD180 Ligation Fail to Respond to Anti IgM Stimulation as Well.
    • Farren T, Liu FT, Stephens C, Macey M, Jenner M, Prentice A, Nathwani A, Agrawal S (2010). Inter Laboratory Assessment of Minimal Residual Disease In Chronic Lymphocytic Leukemia - Evaluation of the Novel CD160FCA Assay.
    • 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.
    • Porakishvili N, Lydyard P, Bremser A, Vispute K, Memon A, Kulikova N, Clarke E, Rai KR, Nathwani A, Damle RN, Chiorazzi N (2010). B-Cell Chronic Lymphocytic Leukemia (B-CLL) Cells Unresponsive to CD180 Ligation Fail to Respond to Anti-IgM Stimulation as Well.
    • Nathwani A (2010). Haemophilia B gene therapy study in the UK. HAEMOPHILIA, 16(6), 973 - 973.
    • Nathwani AC, Tuddenham EGD, Rosales C, McIntosh J, Riddell A, Rustagi P, Galder B, Kay M, Allay J, Coleman J, Sleep S, High KA, Mingozzi F, Gray J, Reiss U, Nienhuis AW, Davidoff AM (2010). Early clinical trial results following administration of a low dose of a novel self complementary adenohyphen;associated viral vector encoding human factor ix in two subjects with severe Haemophilia B.
    • Hanley J, Rastegarlari G, Nathwani AC (2010). An introduction to induced pluripotent stem cells. Br.J.Haematol., 151(1), 16 - 24. doi:10.1111/j.1365-2141.2010.08296.x
    • Hillmen P, Gribben JG, Follows GA, Milligan DW, Sayala HA, Moreton P, Oscier D, Dearden CE, Kennedy DB, Pettitt AR, Nathwani A, Cohen D, Gregory WM, Rawstron AC, Hayward CRW, Pocock C (2010). An open-label phase II study to investigate the safety and efficacy of rituximab plus chlorambucil in previously untreated patients with CD20-positive B-cell chronic lymphocytic leukaemia (CLL): On behalf of the NCRI CLL sub-group.
    • Mattar CN, Nathwani AC, Dighe N, Waddington S, McIntosh J, Biswas A, Fisk N, Davidoff AM, David A, Peebles D, Nowrouzi A, Kappel C, Schmidt M, Choolani M, Chan J (2010). Late-Gestation Intravascular Delivery of Self-Complementary AAV Leads to Sustained Transgene Expression with the Possibility of Vector Integration Events in the Liver.
    • 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.
    • Tan MH, Smith AJ, Pawlyk B, Xu XY, Liu XQ, Bainbridge JB, Basche M, McIntosh J, Tran HV, Nathwani A, Li TS, Ali RR (2010). Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors (vol 18, pg 2099, 2009). HUM MOL GENET, 19(4), 735 - 735. doi:10.1093/hmg/ddp540
    • Walton JA, Lydyard PM, Nathwani A, Emery V, Akbar A, Glennie MJ, Porakishvili N (2010). Patients with B cell chronic lymphocytic leukaemia have an expanded population of CD4 perforin expressing T cells enriched for human cytomegalovirus specificity and an effector-memory phenotype.. Br J Haematol, 148(2), 274 - 284. doi:10.1111/j.1365-2141.2009.07964.x
    • Tracey L, Streck CJ, Du Z, Williams RF, Pfeffer LM, Nathwani AC, Davidoff AM (2010). NF-kappa B activation mediates resistance to IFN beta in MLL-rearranged acute lymphoblastic leukemia. LEUKEMIA, 24(4), 806 - 812. doi:10.1038/leu.2010.2
    • 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.
    • Hillmen P, Gribben JG, Follows GA, Milligan D, Sayala HA, Moreton P, Oscier DG, Dearden CE, Kennedy DB, Pettitt AR, Nathwani A, Cohen D, Rawstron A, Pocock CFE (2010). Rituximab Plus Chlorambucil In Patients with CD20-Positive B-Cell Chronic Lymphocytic Leukemia (CLL): Final Response Analysis of An Open-Label Phase II Study.
    • Sims TL, McGee M, Williams RF, Myers AL, Tracey L, Hamner JB, Ng C, Davidoff AM, Wu J, McCarville B, Gaber MW, Nathwani AC (2010). IFN-β restricts tumor growth and sensitizes alveolar rhabdomyosarcoma to ionizing radiation. Molecular Cancer Therapeutics, 9(3), 761 - 771. doi:10.1158/1535-7163.MCT-09-0800
    • Nathwani A, Tuddenham E, Rosales C, McIntosh J, Riddell A, Rustagi P, Glader B, Kay M, Allay J, Coleman J, Sleep S, High KA, Mingozzi F, Gray JT, Reiss UM, Nienhuis AW, Davidoff A (2010). Early Clinical Trial Results Following Administration of a Low Dose of a Novel Self Complementary Adeno Associated Viral Vector Encoding Human Factor IX In Two Subjects with Severe Hemophilia B.
    • Nathwani A, Tuddenham E, Rosales C, McIntosh J, Riddell A, Rustagi P, Glader B, Kay M, Allay J, Coleman J, Sleep S, High KA, Mingozzi F, Gray JT, Reiss UM, Nienhuis AW, Davidoff A (2010). Early Clinical Trial Results Following Administration of a Low Dose of a Novel Self Complementary Adeno-Associated Viral Vector Encoding Human Factor IX In Two Subjects with Severe Hemophilia B.
    • McIntosh J, Lenting PJ, Tuddenham E, Sotannde M, Waddington S, Gray JT, Davidoff A, Nathwani A (2010). Stable High Level Coagulation Factor VIII Expression In Vivo Following Gene Transfer Using a Novel Expression Cassette Encoding a More Potent FVIII Variant.
    • McIntosh J, Lenting PJ, Tuddenham E, Sotannde M, Waddington S, Gray JT, Davidoff A, Nathwani A (2010). Stable High Level Coagulation Factor VIII Expression In Vivo Following Gene Transfer Using a Novel Expression Cassette Encoding a More Potent FVIII Variant.
    • Farren T, Liu FT, Stephens C, Macey M, Jenner M, Prentice A, Nathwani A, Agrawal S (2010). Inter-Laboratory Assessment of Minimal Residual Disease In Chronic Lymphocytic Leukemia-7 Evaluation of the Novel CD160FCA Assay.
    • Williams RF, Sims TL, Tracey L, Myers AL, Ng CY, Poppleton H, Nathwani AC, Davidoff AM (2010). Maturation of tumor vasculature by interferon-beta disrupts the vascular niche of glioma stem cells. Anticancer Res., 30(9), 3301 - 3308.
    • Williams RF, Myers AL, Sims TL, Ng CY, Nathwani AC, Davidoff AM (2010). Targeting multiple angiogenic pathways for the treatment of neuroblastoma. J.Pediatr.Surg., 45(6), 1103 - 1109. doi:10.1016/j.jpedsurg.2010.02.073
    • Georgiadis A, Tschernutter M, Bainbridge JW, Robbie SJ, McIntosh J, Nathwani AC, Smith AJ, Ali RR (2010). AAV-mediated knockdown of peripherin-2 in vivo using miRNA-based hairpins. Gene Ther., 17(4), 486 - 493. doi:10.1038/gt.2009.162
    • McGee MC, Hamner JB, Williams RF, Rosati SF, Sims TL, Ng CY, Gaber MW, Calabrese C, Wu J, Nathwani AC, Duntsch C, Merchant TE, Davidoff AM (2010). Improved intratumoral oxygenation through vascular normalization increases glioma sensitivity to ionizing radiation. Int.J.Radiat.Oncol.Biol.Phys., 76(5), 1537 - 1545. doi:10.1016/j.ijrobp.2009.12.010
    • Hillmen P, Gribben JG, Follows GA, Milligan D, Sayala HA, Moreton P, Oscier DG, Deanden CE, Kennedy DB, Pettitt AR, Nathwani A, Cohen D, Rawstron A, Pocock CFE (2010). Rituximab Plus Chlorambucil In Patients with CD20-Positive B Cell Chronic Lymphocytic Leukemia (CLL) Final Response Analysis of An Open Label Phase H Study.

    2009

    • Hillmen P, Gribben JG, Follows GA, Milligan DW, Sayala HA, Moreton P, Oscier D, Dearden CE, Kennedy DB, Pettitt AR, Nathwani A, Cohen D, Gregory WM, Rawstron AC, Hayward CRW, Pocock C (2009). An Open-Label Phase II Study to Investigate the Safety and Efficacy of Rituximab Plus Chlorambucil in Previously Untreated Patients with CD20-Positive B-Cell Chronic Lymphocytic Leukaemia (CLL)..
    • Mattar CN, Dighe NM, Waddington SN, McIntosh J, Biswas A, Fisk NM, Davidoff AM, Nathwani AC, Choolani MA, Chan J (2009). Long-Term Transgene Expression in New-Born Cynomolgus Macaques Following Intra-Uterine Gene Transfer of Self-Complementary AAV.
    • 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.
    • Shih CS, Laurie N, Holzmacher J, Spence Y, Nathwani AC, Davidoff AM, Dyer MA (2009). AAV-mediated local delivery of interferon-beta for the treatment of retinoblastoma in preclinical models. Neuromolecular.Med., 11(1), 43 - 52. doi:10.1007/s12017-009-8059-0
    • Kirwan M, Beswick R, Vulliamy T, Nathwani AC, Walne AJ, Casimir C, Dokal I (2009). Exogenous TERC alone can enhance proliferative potential, telomerase activity and telomere length in lymphocytes from dyskeratosis congenita patients. Br.J.Haematol., 144(5), 771 - 781. doi:10.1111/j.1365-2141.2008.07516.x
    • Nathwani AC, Cochrane M, McIntosh J, Ng CYC, Zhou J, Gray JT, Davidoff AM (2009). Enhancing transduction of the liver by adeno-associated viral vectors. GENE THER, 16(1), 60 - 69. doi:10.1038/gt.2008.137
    • Reiss U, Davidoff A, Gray J, Nienhuis A, Nathwani A (2009). Dose response results of self complementary adeno-associated virus (AAV) vector-mediated factor IX gene transfer in non-human primates. HAEMOPHILIA, 15(2), 635 - 635.

    2008

    • 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. Hum.Gene Ther., 19(10), 1050 - 1056.
    • Delgado J, Pillai S, Benjamin R, Caballero D, Martino R, Nathwani A, Lovell R, Thomson K, Perez-Simon JA, Sureda A, Kottaridis P, Vazquez L, Peggs K, Sierra J, Milligan D, Mackinnon S (2008). The effect of in vivo T cell depletion with alemtuzumab on reduced-intensity allogeneic hematopoietic cell transplantation for chronic lymphocytic leukemia.. Biology of Blood and Marrow Transplantation, 14(11), 1288 - 1297.
    • Buch PK, McIntosh J, Georgiadis A, Mowat F, Tschernutter M, Smith AJ, Nathwani AC, Ali RR (2008). Pseudotyped adeno-associated viral vectors to enhance gene replacement therapy in a mouse model of retinal degeneration.
    • 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.
    • Evans A, Riva A, Cooksley H, Phillips S, Nathwani A, Craigen J, Brett S, Chokshi S, Naoumov N (2008). Longitudinal analysis of PD1 expression: Impact of HBeAg seroconversion during antiviral treatment of chronic hepatitis B.
    • Osman E, Evans V, Graham IR, Athanasopoulos T, McIntosh J, Nathwani AC, Simons JP, Dickson GO, J S (2008). Preliminary evaluation of a self-complementary AAV2/8 vector for hepatic gene transfer of human apoE3 to inhibit atherosclerotic lesion development in apoE-deficient mice. Atherosclerosis, 204(1), 121 - 126. doi:10.1016/j.atherosclerosis.2008.08.043
    • Kyriakou C, Rabin N, Pizzey A, Nathwani A, Yong K (2008). Factors that influence short-term homing of human bone marrow-derived mesenchymal stem cells in a xenogeneic animal model. Haematologica, 93(10), 1457 - 1465.
    • Carlisle RC, Benjamin R, Briggs SS, Sumner-Jones S, McIntosh J, Gill D, Hyde S, Nathwani A, Subr V, Ulbrich K, Seymour LW, Fisher KD (2008). Coating of adeno-associated virus with reactive polymers can ablate virus tropism, enable retargeting and provide resistance to neutralising antisera. The Journal of Gene Medicine, 10(4), 400 - 411.
    • Graham T, McIntosh J, Work LM, Nathwani A, Baker AH (2008). Performance of AAV8 vectors expressing human factor IX from a hepatic-selective promoter following intravenous injection into rats.. Genet Vaccines Ther, 6, 9 - . doi:10.1186/1479-0556-6-9
    • David AL, McIntosh J, Nathwani A, Cook T, Boyd M, Weisz B, Wigley V, Abi Nader K, Rodeck CH, Peebles DM (2008). Long-term expression of human factor IX after ultrasound-guided delivery of AAV8 hFIX to fetal sheep in utero.
    • Rosati SF, Williams RF, Nunnally LC, McGee MC, Sims TL, Tracey L, Zhou J, Ng CY, Davidoff AM, Stewart CF, Fan M, Pfeffer LM, Nathwani AC (2008). IFN-β sensitizes neuroblastoma to the antitumor activity of temozolomide by modulating O-methylguanine DNA methyltransferase expression. Molecular Cancer Therapeutics, 7(12), 3852 - 3858. doi:10.1158/1535-7163.MCT-08-0806
    • Nathwani A, Rosati SF, Williams RF, Nunnally LC, McGee MC, Sims TL, Tracey L, Zhou J, Fan M, Ng CY, Stewart CF, Pfeffer LM, Davidoff AM (2008). IFN-beta sensitizes neuroblastoma to the antitumor activity of temozolomide by modulating O6-methylguanine DNA methyltransferase expression.. Mol Cancer Ther, 7(12), 3852 - 3858.
    • Nathwani A, Quinn JP, Mohamedbhai S, Chipperfield K, Treacy M, D'Sa S (2008). Efficacy of rituximab in combination with steroids in refractory chronic lymphocytic leukemia.. Leuk Lymphoma, 49(10), 1995 - 1998.
    • David AL, McIntosh J, Weisz B, Boyd M, Cook T, Wigley V, Kymalainen H, Abi-Nader K, Rodeck CH, Peebles DM, Nathwani A (2008). Long term perinatal gene transfer after clinically applicable delivery of prenatal gene therapy in the sheep.
    • David AL, McIntosh J, Nathwani A, Cook T, Boyd M, Weisz B, Wigley V, Abi-Nader K, Rodeck C, Peebles D (2008). Long term expression of human factor IX after ultrasound-guided fetal gene therapy in sheep.
    • Kyriakou C, Rabin N, Pizzey A, Nathwani A, Yong K (2008). Short term homing of human bone marrow derived mesenchymal stem cells (hMSCs) in a xenogeneic animal model is influenced by animal age and is increased by enforced expression of CXCR4. Haematologica, 93(10), 1457 - 1465.
    • Evans A, Riva A, Cooksley H, Phillips S, Puranik S, Nathwani A, Brett S, Chokshi S, Naoumov NV (2008). Programmed death 1 expression during antiviral treatment of chronic hepatitis B: Impact of hepatitis B e-antigen seroconversion. Hepatology, 48(3), 759 - 769.
    • Natkunarajah M, Trittibach P, McIntosh J, Duran Y, Barker SE, Smith AJ, Nathwani AC, Ali RR (2008). Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8. Gene Therapy, 15(6), 463 - 467.
    • Graham T, McIntosh J, Work LM, Nathwani A, Baker AH (2008). Performance of AAV8 vectors expressing human factor IX from a hepatic-selective promoter following intravenous injection into rats. Genetic Vaccines and Therapy, 3, 6 - 9.
    • Quinn J, Mohamedbhai S, Treacy M, D'Sa S, Nathwani A (2008). Rituximab in combination with high-dose steroids: a safe and effective salvage regimen in advanced, refractory chronic lymphocytic leukaemia.

    2007

    • Nathwani A, Dickson PV, Hamner JB, Streck CJ, Ng CY, McCarville MB, Calabrese C, Gilbertson RJ, Stewart CF, Wilson CM, Gaber MW , Pfeffer LM, Skapek SX, Davidoff AM (2007). Continuous delivery of IFN-beta promotes sustained maturation of intratumoral vasculature.. Mol Cancer Res, 5(6), 531 - 542.
    • Nathwani AC, Gray JT, McIntosh J, Ng CY, Zhou J, Spence Y, Cochrane M, Gray E, Tuddenham EG, Davidoff AM (2007). Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates.. Blood, 109(4), 1414 - 1421. doi:10.1182/blood-2006-03-010181
    • Benjamin R, Khwaja A, Singh N, McIntosh J, Meager A, Wadhwa M, Streck C, Ng C, Davidoff AM, Nathwan AC (2007). Continuous delivery of human type I interferons (α/β)has significant activity against acute myeloid leukemia cells in vitro and in a xenograft model. Blood, 109(3), 1244 - 1247.
    • Rabin N, Kyriakou C, Coulton L, Gallagher OM, Buckle C, Benjamin R, Singh N, Glassford J, Otsuki T, Nathwani AC, Croucher PI, Yong KL (2007). A new xenograft model of myeloma bone disease demonstrating the efficacy of human mesenchymal stem cells expressing osteoprotegerin by lentiviral gene transfer.. Leukemia, , - .
    • Benjamin R, Singh N, McIntosh J, Meager A, Wadhwa M, Streck C, Ng C, Davidoff AM, Nathwani AC (2007). Continuous delivery of human type I interferons ({alpha}/{beta}) has significant activity against acute myeloid leukemia cells in vitro and in a xenograft model.. Blood, 109(3), 1244 - 1247.
    • Vaessen SFC, Comijn L, Snapper J, Nathwani AC, Kastelein J, Petry H, Kuivenhoven JA, Twisk J (2007). Correction of HDL deficiency in ApoA-I-deficient mice following self-complementary AAV-mediated liver expression of human ApoA-I.
    • Rabin N, Coulton L, Gallagher OM, Benjamin R, Singh N, McIntosh JH, Buckle C, Nathwani A, Croucher P, Yong K (2007). Micro-computer tomography (MICROCT) analysis demonstrates extensive bone loss in the KMS-12-BM xenogeneic murine model of myeloma.
    • Quinn J, Mohamedbhai S, Treacey M, D'Sa S, Nathwani A (2007). Rituximab in combination with high-dose steroids in advanced, refractory chronic lymphocytic leukaemia.
    • Barkley LR, Hong HK, Kingsbury SR, McCormick LA, James M, Nathwani AC, Stoeber K, Williams GH (2007). Cdc6 controls proliferative capacity during establishment of the differentiated state. Experimental Cell Research, 313(17), 3789 - 3799. doi:10.1016/j.yexcr.2007.07.004
    • 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.
    • Osman E, Graham I, Athanasopoulos T, Nathwani AC, Owen JS (2007). Liver-directed gene transfer of atheroprotective human ApoA-I-Milano.
    • Riva A, Paulon E, Nathwani A, Taylor J, Naoumov NV (2007). Adenovirus-mediated delivery of HBV DNA in dendritic cells: A potent approach to overcome impaired T-cell reactivity in chronic hepatitis B.

    2006

    • Kyriakou C, Rabin N, Nathwani A, Pizzey A, Linch DC, Yong K (2006). Factors influencing short term in vivo biodistribution of human bone marrow derived mesenchymal stem cells (hMSCs) in a xenogeneic animal model..
    • Rabin N, Kyriakou C, Benjamin R, Pizzey A, Gallagher O, Singh N, Buckle C, Croucher P, Nathwani A, Yong K (2006). Gene modified human mesenchymal stem cells expressing osteoprotegerin inhibit osteoclast activation and increase trabecular bone area in a xenogeneic murine model of myeloma..
    • Benjamin R, Davidoff AM, Singh N, Mcintosh J, Khwaja A, Streck C, Ng C, Meager A, Wadhwa M, Nathwani AC (2006). Therapeutic efficacy of interferon-beta in a xenograft model of acute myeloid leukaemia.
    • Streck CJ, Dickson PV, Ng CY, Zhou J, Hall MM, Gray JT, Nathwani AC, Davidoff AM (2006). Antitumor efficacy of AAV-mediated systemic delivery of interferon-beta.. Cancer Gene Therapy, 13(1), 99 - 106.
    • McCarville MB, Streck CJ, Dickson PV, Davidoff AM, Li C-S, Nathwani AC (2006). Angiogenesis inhibitors in a murine neuroblastoma model: Quantitative assessment of intratumoral blood flow with contrast-enhanced gray-scale US. Radiology, 240(1), 73 - 81. doi:10.1148/radiol.2401050709
    • McCarville MB, Streck CJ, Dickson PV, Li CS, Nathwani AC, Davidoff AM (2006). Angiogenesis inhibitors in a murine neuroblastoma model: quantitative assessment of intratumoral blood flow with contrast-enhanced gray-scale US.. Radiology, 240(1), 73 - 81.
    • Nathwani AC, Gray JT, Ng CY, Zhou J, Spence Y, Waddington SN, Tuddenham EG, Kemball-Cook G, McIntosh J, Boon-Spijker M, Mertens K, Davidoff AM (2006). Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver.. Blood, 107(7), 2653 - 2661.
    • Mcintosh J, Cochrane M, Tuddenham EGD, Davidoff AM, Nathwani AC (2006). Expression of human FIX at therapeutic levels in nonhuman primates following a single peripheral vein infusion of low doses of self complementary AAV vector renews hope for haemophilia B gene therapy.
    • Kyriakou CA, Yong KL, Benjamin R, Pizzey A, Dogan A, Singh N, Davidoff AM, Nathwani AC (2006). Human mesenchymal stem cells (hMSCs) expressing truncated soluble vascular endothelial growth factor receptor (tsFlk-1) following lentiviral-mediated gene transfer inhibit growth of Burkitt's lymphoma in a murine model.. The Journal of Gene Medicine, 8(3), 253 - 264.

    2005

    • Jewell AP, Cochrane M, McIntosh J, Benjamin R, Nathwani A (2005). Comparison of viral vectors for gene transfer into CLL cells: Efficient transduction with adeno-associated virus-8 (AAV-8)..
    • Nathwani AC, Davidoff AM, Linch DC (2005). A review of gene therapy for haematological disorders.. British Journal of Haematology, 128(1), 3 - 17.
    • Davidoff AM, Ng CY, Zhang Y, Streck C, Mabry S, Barton S, Baudino T, Zhou J, Kerbel R, Vanin E, Nathwani AC (2005). Careful decoy receptor titering is required to inhibit tumor angiogenesis while avoiding adversely altering VEGF bioavailability.. Molecular Therapy, 11(2), 300 - 310. doi:10.1016/j.ymthe.2004.09.014
    • Davidoff AM, Gray JT, Ng CY, Zhang Y, Zhou J, Spence Y, Bakar Y, Nathwani AC (2005). Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models.. Molecular Therapy, 11(6), 875 - 888.
    • Nathwani AC, McIntosh J, Davidoff AM (2005). An update on gene therapy for hemophilia.. Current Hematology Reports, 4(4), 287 - 293.
    • Dickson PV, Nathwani AC, Davidoff AM (2005). Delivery of antiangiogenic agents for cancer gene therapy.. Technology in Cancer Research and Treatment, 4(4), 331 - 341.
    • Streck CJ, Dickson PV, Ng CY, Zhou J, Gray JT, Nathwani AC, Davidoff AM (2005). Adeno-associated virus vector-mediated systemic delivery of IFN-beta combined with low-dose cyclophosphamide affects tumor regression in murine neuroblastoma models.. Clinical Cancer Research, 11(16), 6020 - 6029.
    • Streck CJ, Ng CY, Zhang Y, Zhou J, Nathwani AC, Davidoff AM (2005). Interferon-mediated anti-angiogenic therapy for neuroblastoma.. Cancer Letters, 228(1), 163 - 170.
    • Manoussaka MS, Memon A, Nathwani A, Porakishvili N, Clark E, Lydyard PM (2005). Toll like receptors (TLRs) in B cell chronic lymphocytic leukemia (B-CLL)..
    • Walton J, Lawson K, Manoussaka MS, Nathwani A, Emory V, Lydyard PM, Porakishvili N (2005). CD4(+) perforin(+) T cells in B-CLL: MHC class II restriction and anti-cytomegalovirus reactivity..
    • Streck CJ, Zhang Y, Zhou J, Ng C, Nathwani AC, Davidoff AM (2005). Adeno-associated virus vector-mediated delivery of pigment epithelium-derived factor restricts neuroblastoma angiogenesis and growth.. Journal of Pediatric Surgery, 40(1), 236 - 243.
    • Porakishvili N, Kulikova N, Jewell AP, Youinou PY, Nedelec S, Yong K, Nathwani A, Heelan B, Duke V, Hamblin TJ, Wallace P, Ely P, Clark EA, Lydyard PM (2005). CD180 is more highly expressed by B cell chronic lymphocytic leukaemia (B-CLL) cells using IgVh mutated than those using IgVh unmutated genes. British Journal of Haematology, 131(3), 313 - 319.

    2004

    • Davidoff AM, Ng CYC, Sleep S, Gray J, Azam S, Zhao Y, McIntosh JH, Karimipoor M, Nathwani AC (2004). Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock. J VIROL METHODS, 121(2), 209 - 215. doi:10.1016/j.jviromet.2004.07.001
    • Porakishvili N, Manoussaka M, Kulikova N, Walton J, Nathwani A, Jewell AP, Stevenson F, Clark E, Lydyard PM (2004). Activation and proliferation of B cell chronic lymphocytic leukaemia (B-CLL) cells through anti-CD180, Anti-CD40 and IL-4..
    • Streck CJ, Zhang Y, Miyamoto R, Zhou J, Ng CYC, Nathwani AC, Davidoff AM (2004). Restriction of neuroblastoma angiogenesis and growth by interferon-alpha/beta. doi:10.1016/j.surg.2004.04.014
    • Streck CJ, Zhou J, Ng CY, Zhang Y, Nathwani AC, Davidoff AM (2004). Longterm recombinant adeno-associated, virus-mediated, liver-generated expression of an angiogenesis inhibitor improves survival in mice with disseminated neuroblastoma.. Journal of the American College of Surgeons, 199(1), 78 - 86.
    • Gray JT, Davidoff AM, Nathwani AC (2004). A novel human factor IX expression cassette packaged as self-complementary DNA dimers in adeno-associated virus vectors results in dramatically improved liver transduction, significantly improving prospects for hemophilia B gene therapy.
    • Nathwani AC, Benjamin R, Nienhuis AW, Davidoff AM (2004). Current status and prospects for gene therapy. VOX SANG, 87(2), 73 - 81.
    • Davidoff AM, Nathwani AC (2004). Antiangiogenic gene therapy for cancer treatment.. Current Hematology Reports, 3(4), 267 - 273.
    • Lajmi AR, Sleep S, Ng C, Zhou JF, Spence YY, Davidoff AM, Nathwani AC (2004). Scaleable membrane chromatography purification method for adeno-associated viral vectors..
    • Folarin NI, Baker RJ, Duke V, Yogashangary BC, Vadikolia C, Wickremasinge RG, Casanova AA, Lowdell MW, Hoffbrand AV, Nathwani A, Virchis AE, Mehta AB, Foroni L, Ganeshaguru K (2004). Zap-70 and IgVH mutation status in combination with p53 functional analysis identifies the response of poor prognosis B-CLL patients to conventional cytotoxic agents.
    • Streck CJ, Zhang Y, Miyamoto R, Zhou J, Ng CY, Nathwani AC, Davidoff AM (2004). Restriction of neuroblastoma angiogenesis and growth by interferon-alpha/beta.. Surgery, 136(2), 183 - 189.
    • Nivsarkar MS, Nathwani A, Mosley K, Davies N, Gregory LG, David AL, Themis M, Coutelle C, Waddington SN (2004). Testosterone induced increase in transgene expression in vivo using Adenoviral Gene Delivery Vectors.
    • Porakishvili N, Kulikova N, Manoussaka M, Jewell AP, Youinou PY, Nedelec S, Yong K, Nathwani A, Heelan B, Hamblin T, Wallace P, Ely P, Clarke E, Lydyard PM (2004). Differential expression of CD180 and sIgM by B chronic lymphocytic leukaemia cells (B-CLL) using mutated and unmutated IgVh genes..
    • Walton J, Lawson K, Manoussaka MS, Nathwani A, Emory V, Lydyard PM, Porakishvili N (2004). Perforin expressing CD4+T cells respond to CMV antigens in CMV seropositive patients with B cell chronic lymphocytic leukaemia (B-CLL)..
    • Nathwani AC, Davidoff AM, Tuddenham EG (2004). Prospects for gene therapy of haemophilia.. Haemophilia, 10(4), 309 - 318.

    2003

    • Ng CY, Karimipoor M, Zhou JF, Lajmi A, Zhong WY, Spence Y, Davidoff AM, Nathwani AC (2003). Highly pure recombinant adeno-associated viral (rAAV) vectors based on serotype 8 generated by a simple scalable chromatography method efficiently transduce murine liver..
    • McCormick AL, Benjamin R, Pizzey A, Kyriakou C, Nathwani AC (2003). Facilitation of lentivirus mediated gene transfer into primary human and murine progenitors by Antp peptide.
    • Kyriakou CA, Yong KL, Benjamin R, Gandini D, Davidoff AM, Nathwani AC (2003). Human mesenchymal stem cells (hMSCs) expressing truncated soluble vascular endothelial growth factor receptor (tsFlk-1) following lentiviral mediated gene transfer inhibit growth of Burkitt's lymphoma in murine models..
    • Nathwani AC, Ng CY, Zhou JF, Spence Y, Nienhuis AW, Davidoff AM (2003). Liver targeted delivery of recombinant adeno-associated virus (rAAV) serotype 5 vectors leads to sustained therapeutic expression of human factor IX (hFIX) in non-human primates previously sensitized to rAAV2 vectors..
    • Porakishvili N, Kulikova N, Jewell AP, Youinou PY, Yong K, Nathwani A, Heelan B, Duke V, Stevenson F, Hamblin TJ, Wallace P, Ely P, Clark EA, Lydyard PM (2003). Preferential expression of CD180 by B cell chronic lymphocytic leukaemia (B-CLL) cells using IgVh mutated versus those using IgVh unmutated genes.
    • Benjamin R, Davidoff AM, Ng CYC, Pizzey A, Yong K, Nathwani AC (2003). Murine models of hematological malignancies that mimic the disease phenotype in humans..
    • Porakishvili N, Slovick A, Jewell AP, Youinou PY, Nathwani A, Yong K, Stevenson F, Clark EA, Lydyard PM (2003). Activation of B-CLL cells by anti CD180 monoclonal antibodies: Responder and non-responder patients..
    • Davidoff AM, Ng CY, Zhou J, Spence Y, Nathwani AC (2003). Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway.. Blood, 102(2), 480 - 488. doi:10.1182/blood-2002-09-2889

    2002

    • Davidoff AM, Nathwani AC, Spurbeck WW, Ng CY, Zhou J, Vanin EF (2002). rAAV-mediated long-term liver-generated expression of an angiogenesis inhibitor can restrict renal tumor growth in mice.. Cancer Research, 62(11), 3077 - 3083.
    • Nathwani A, Davidoff AM, Hanawa H, Hu Y, Hoffer FA, Nikanorov A, Slaughter C, Ng CY, Zhou J, Lozier JN, Mandrell TD, Vanin EF, Nienhuis AW (2002). Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques.. Blood, 100(5), 1662 - 1669. doi:10.1182/blood-2002-02-0589
    • Hanawa H, Kelly PF, Nathwani AC, Persons DA, Vandergriff JA, Hargrove P, Vanin EF, Nienhuis AW (2002). Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood.. Molecular Therapy, 5(3), 242 - 251.
    • Forbes LV, Gale RE, Pizzey A, Pouwels K, Nathwani A, Linch DC (2002). An activating mutation in the transmembrane domain of the granulocyte colony-stimulating factor receptor in patients with acute myeloid leukemia.. Oncogene, 21(39), 5981 - 5989. doi:10.1038/sj.onc.1205767

    2001

    • Kelly PF, Carrington J, Nathwani A, Vanin EF (2001). RD114-pseudotyped oncoretroviral vectors. Biological and physical properties.. Ann N Y Acad Sci, 938, 262 - 276.
    • Nathwani A, Davidoff A, Hanawa H, Zhou JF, Vanin EF, Nienhuis AW (2001). Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA.. Blood, 97(5), 1258 - 1265.

    2000

    • Nathwani AC, Hanawa H, Vandergriff J, Kelly P, Vanin EF, Nienhuis AW (2000). Efficient gene transfer into human cord blood CD34+ cells and the CD34+CD38- subset using highly purified recombinant adeno-associated viral vector preparations that are free of helper virus and wild-type AAV.. Gene Therapy, 7(3), 183 - 195.
    • Kelly PF, Vandergriff J, Nathwani A, Nienhuis AW, Vanin EF (2000). Highly efficient gene transfer into cord blood nonobese diabetic/severe combined immunodeficiency repopulating cells by oncoretroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein.. Blood, 96(4), 1206 - 1214.

    1999

    • Nathwani AC, Persons DA, Stevenson SC, Frare P, McClelland A, Nienhuis AW, Vanin EF (1999). Adenovirus-mediated expresssion of the murine ecotropic receptor facilitates transduction of human hematopoietic cells with an ecotropic retroviral vector.. Gene Therapy, 6(8), 1456 - 1468.

    1996

    • Durosinmi MA, Matutes E, Ryan K, Nathwani A, Hedges M, Catovsky D (1996). Royal Marsden teaching cases. An elderly man with weight loss and anaemia.. Leukemia and Lymphoma, 23(1), 181 - 183.

    1994

    • Nathwani AC, Gale KM, Pemberton KD, Crossman DC, Tuddenham EG, McVey JH (1994). Efficient gene transfer into human umbilical vein endothelial cells allows functional analysis of the human tissue factor gene promoter.. British Journal of Haematology, 88(1), 122 - 128.

    1993

    • Nathwani AC, Goldman JM (1993). Management of chronic myeloid leukaemia in lymphoid blast transformation.. Haematologica, 78(3), 162 - 166.

    1992

    • Nathwani AC, Tuddenham EG (1992). Epidemiology of coagulation disorders.. Baillière's Clinical Haematology, 5(2), 383 - 439.

    1991

    • Vulliamy TJ, Othman A, Town M, Nathwani A, Falusi AG, Mason PJ, Luzzatto L (1991). Polymorphic sites in the African population detected by sequence analysis of the glucose-6-phosphate dehydrogenase gene outline the evolution of the variants A and A-.. Proc Natl Acad Sci U S A, 88(19), 8568 - 8571.
    • Vulliamy TJ, Othman A, Town M, Nathwani A, Falusi Y, Luzzatto L (1991). Linkage disequilibrium of polymorphic sites in the G6PD gene in African populations and the origin of G6PD A-.. Gene Geography, 5(1), 13 - 21.

    1990

    • Warren AJ, Hegde UM, Nathwani A, Reilly IA (1990). Systemic vasculitis and myelodysplasia.. British Journal of Haematology, 75(4), 627 - 629.

    • Tuddenham EGD, Nathwani AC, Rangarajan S, Rosales C, McIntosh J, Linch DC, Chowdary P, Riddell A, Pie J, Harrington C, O'Beirne J, Smith K, Pasi J, Glader B, Rustagi P, Ng CYC, Kay MA, Zhou J, Spence Y, Morton CL, Allay J, Coleman J, Sleep S, Cunningham JM, Srivastava D, Basner-Tschakarjan E, Mingozzi F, High KA, Gray JT, Reiss UM, Nienhuis AW, Davidoff AM (). Adeno-Associated Viral Vector Mediated Gene Transfer for Hemophilia B.. N Engl J Med, 365, - .
    • Nathwani AC, Cochrane M, McIntosh J, Ng CY, Zhou J, Gray JT, Davidoff AM (). Enhancing transduction of the liver by adeno-associated viral vectors. Gene Therapy, , - .