A C Nathwani

Summary

Affiliation: University College London
Country: UK

Publications

  1. ncbi request reprint A review of gene therapy for haematological disorders
    Amit C Nathwani
    Department of Haematology, University College London, London, UK
    Br J Haematol 128:3-17. 2005
  2. ncbi request reprint An update on gene therapy for hemophilia
    Amit C Nathwani
    Department of Haematology, University College London, 98 Chenies Mews, London WC1E 6HX, UK
    Curr Hematol Rep 4:287-93. 2005
  3. ncbi request reprint Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood
    Hideki Hanawa
    Department of Hematology Oncology, Division of Experimental Hematology, St Jude Children s Research Hospital, Memphis, Tennessee, USA
    Mol Ther 5:242-51. 2002
  4. ncbi request reprint 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
    Amit C Nathwani
    Department of Haematology, University College London, United Kingdom
    Blood 100:1662-9. 2002
  5. pmc Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
    Amit C Nathwani
    Department of Haematology, University College London Cancer Institute, London, United Kingdom
    N Engl J Med 365:2357-65. 2011
  6. ncbi request reprint Prospects for gene therapy of haemophilia
    A C Nathwani
    Department of Haematology, University College London, 98 Chenies Mews, London, WC1E 6HX, UK
    Haemophilia 10:309-18. 2004
  7. ncbi request reprint Current status of gene therapy for hemophilia
    Amit C Nathwani
    Department of Haematology, University College London, 98 Chenies Mews, London WCIE 6HX, UK
    Curr Hematol Rep 2:319-27. 2003
  8. pmc 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
    Amit C Nathwani
    Department of Haematology, University College London, 98 Chenies Mews, London, United Kingdom, WC1E 6HX
    Blood 107:2653-61. 2006
  9. pmc 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
    Amit C Nathwani
    Department of Haemotology, University College London, UK, and Department of Surgery, St Jude Children s Research Hospital, Memphis, TN, USA
    Blood 109:1414-21. 2007
  10. pmc Enhancing transduction of the liver by adeno-associated viral vectors
    A C Nathwani
    Department of Haematology, UCL Cancer Institute, London, UK
    Gene Ther 16:60-9. 2009

Collaborators

Detail Information

Publications36

  1. ncbi request reprint A review of gene therapy for haematological disorders
    Amit C Nathwani
    Department of Haematology, University College London, London, UK
    Br J Haematol 128:3-17. 2005
    ..This article reviews the current status of gene therapy and outlines the challenges faced by this emerging technology that holds so much promise for many suffering from catastrophic disorders...
  2. ncbi request reprint An update on gene therapy for hemophilia
    Amit C Nathwani
    Department of Haematology, University College London, 98 Chenies Mews, London WC1E 6HX, UK
    Curr Hematol Rep 4:287-93. 2005
    ..Although many obstacles still remain, continuing technologic improvements have resulted in impressive advances in this field, which bodes well for patients with hemophilia and other genetic disorders...
  3. ncbi request reprint Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood
    Hideki Hanawa
    Department of Hematology Oncology, Division of Experimental Hematology, St Jude Children s Research Hospital, Memphis, Tennessee, USA
    Mol Ther 5:242-51. 2002
    ....
  4. ncbi request reprint 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
    Amit C Nathwani
    Department of Haematology, University College London, United Kingdom
    Blood 100:1662-9. 2002
    ..This nonhuman-primate model will be useful in further evaluation and development of rAAV vectors for gene therapy of hemophilia B...
  5. pmc Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
    Amit C Nathwani
    Department of Haematology, University College London Cancer Institute, London, United Kingdom
    N Engl J Med 365:2357-65. 2011
    ..Hemophilia B, an X-linked disorder, is ideally suited for gene therapy. We investigated the use of a new gene therapy in patients with the disorder...
  6. ncbi request reprint Prospects for gene therapy of haemophilia
    A C Nathwani
    Department of Haematology, University College London, 98 Chenies Mews, London, WC1E 6HX, UK
    Haemophilia 10:309-18. 2004
    ..The purpose of this review is to outline the current status of gene therapy in light of recent successes and tragedies and to consider the prospects for curing haemophilia in the short-to-medium term...
  7. ncbi request reprint Current status of gene therapy for hemophilia
    Amit C Nathwani
    Department of Haematology, University College London, 98 Chenies Mews, London WCIE 6HX, UK
    Curr Hematol Rep 2:319-27. 2003
    ..Recombinant adeno-associated viral vectors appear most promising for hemophilia gene therapy; however, this review summarizes all the major gene therapy approaches used and outlines the future challenges...
  8. pmc 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
    Amit C Nathwani
    Department of Haematology, University College London, 98 Chenies Mews, London, United Kingdom, WC1E 6HX
    Blood 107:2653-61. 2006
    ..Furthermore, AAV5-pseudotyped scAAV vectors mediated successful transduction in macaques with pre-existing immunity to AAV8. Hence, this novel vector represents an important advance for hemophilia B gene therapy...
  9. pmc 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
    Amit C Nathwani
    Department of Haemotology, University College London, UK, and Department of Surgery, St Jude Children s Research Hospital, Memphis, TN, USA
    Blood 109:1414-21. 2007
    ..These results should make vector administration to patients, especially those with a severe bleeding diathesis, significantly easier and safer...
  10. pmc Enhancing transduction of the liver by adeno-associated viral vectors
    A C Nathwani
    Department of Haematology, UCL Cancer Institute, London, UK
    Gene Ther 16:60-9. 2009
    ..Hence, optimization of individual steps in the AAV gene transfer process can further enhance the potency of AAV-mediated transgene expression, thus increasing the probability of successful gene therapy...
  11. pmc 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
    Amit C Nathwani
    Department of Hematology, University College London Cancer Institute, London, UK
    Mol Ther 19:876-85. 2011
    ..Long-term biochemical, ultrasound imaging, and histologic follow-up of this large cohort of NHP revealed no toxicity. These data support further evaluation of this vector in hemophilia B patients...
  12. ncbi request reprint Current status and prospects for gene therapy
    A C Nathwani
    Department of Haematology, University College London, London, UK
    Vox Sang 87:73-81. 2004
    ..The purpose of this article is to review the current status of gene therapy in light of recent successes and tragedies, and to consider the challenges faced by this relatively new field...
  13. ncbi request reprint Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA
    A C Nathwani
    Division of Experimental Hematology, Department of Hematology/ Oncology, St Jude Children's Research Hospital, Memphis, TN 38105, USA
    Blood 97:1258-65. 2001
    ..Instead, neutralizing anti-hFIX antibodies were detected in all the mice. These observations may have important implications for hemophilia B gene therapy with rAAV vectors...
  14. pmc Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine
    J H McIntosh
    Department of Haematology, UCL Cancer Institute, University College London, Oxford, UK
    Gene Ther 19:78-85. 2012
    ..These observations could significantly improve the prospects of using rAAV vectors for chronic disorders by allowing for repeated vector administration and avoiding the development of antibodies to the transgene product...
  15. ncbi request reprint Maturation of tumor vasculature by interferon-beta disrupts the vascular niche of glioma stem cells
    R F Williams
    Department of Surgery, St Jude Children s Research Hospital, University of Tennessee Health Science Center, Memphis, TN 38105 3678, USA
    Anticancer Res 30:3301-8. 2010
    ..The vascular niche necessary for cancer stem cell maintenance is a potential target for cancer therapy...
  16. doi request reprint AAV-mediated knockdown of peripherin-2 in vivo using miRNA-based hairpins
    A Georgiadis
    Department of Molecular Therapy, Institute of Ophthalmology, University College London, London, UK
    Gene Ther 17:486-93. 2010
    ....
  17. ncbi request reprint Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8
    M Natkunarajah
    Division of Molecular Therapy, UCL Institute of Ophthalmology, University College London, London, UK
    Gene Ther 15:463-7. 2008
    ..This improved transduction efficiency might facilitate the development of improved gene therapy protocols for inherited retinal degenerations, particularly those caused by defects in photoreceptor-specific genes...
  18. ncbi request reprint 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
    Andrew M Davidoff
    Department of Surgery, St Jude Children s Research Hospital, Memphis, TN, USA
    Mol Ther 11:875-88. 2005
    ..In summary, serotype-specific differences in transduction biology and the appreciation of preexisting immunity will likely influence the selection of the rAAV serotype for future clinical trials...
  19. doi request reprint 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
    Eyman Osman
    Department of Medicine, Royal Free and University College Medical School, London, UK
    Atherosclerosis 204:121-6. 2009
    ....
  20. doi request reprint NF-kappaB activation mediates resistance to IFN beta in MLL-rearranged acute lymphoblastic leukemia
    L Tracey
    Department of Surgery, St Jude Children s Research Hospital, Memphis, TN 38105, USA
    Leukemia 24:806-12. 2010
    ..IFN beta combined with agents that inhibit NF-kappaB could have therapeutic potential in the treatment of children with mixed lineage leukemia subtype ALL...
  21. ncbi request reprint rAAV-mediated long-term liver-generated expression of an angiogenesis inhibitor can restrict renal tumor growth in mice
    Andrew M Davidoff
    Department of Surgery, St Jude Children s Research Hospital, 332 North Lauderdale, Memphis, TN 38105, USA
    Cancer Res 62:3077-83. 2002
    ..These findings establish the feasibility of using rAAV vectors in antiangiogenic gene therapy...
  22. ncbi request reprint Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway
    Andrew M Davidoff
    Department of Surgery, St Jude Children s Research Hospital, Memphis, TN 38105, USA
    Blood 102:480-8. 2003
    ..These results have significant implications for gene therapy of autosomal and acquired disorders affecting the liver...
  23. pmc AAV-mediated local delivery of interferon-beta for the treatment of retinoblastoma in preclinical models
    Chie Schin Shih
    Department of Developmental Neurobiology, St Jude Children s Research Hospital, Memphis, TN 38105, USA
    Neuromolecular Med 11:43-52. 2009
    ..These data suggest that AAV-mediated delivery of IFN-beta may provide a complementary approach to systemic chemotherapy which is the standard of care for retinoblastoma around the world...
  24. doi request reprint Exogenous TERC alone can enhance proliferative potential, telomerase activity and telomere length in lymphocytes from dyskeratosis congenita patients
    Michael Kirwan
    Centre for Paediatrics, Institute of Cell and Molecular Science, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, London, UK
    Br J Haematol 144:771-81. 2009
    ..This is the first study of its kind in DC lymphocytes and the first to demonstrate that transduction with TERC alone can improve cell survival and telomere length without the need for exogenous TERT...
  25. ncbi request reprint Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock
    Andrew M Davidoff
    Department of Surgery, St Jude Children s Research Hospital, Memphis, TN, USA
    J Virol Methods 121:209-15. 2004
    ....
  26. ncbi request reprint Longterm recombinant adeno-associated, virus-mediated, liver-generated expression of an angiogenesis inhibitor improves survival in mice with disseminated neuroblastoma
    Christian J Streck
    Department of Surgery, St Jude Children s Research Hospital, Memphis, TN 38105, USA
    J Am Coll Surg 199:78-86. 2004
    ....
  27. ncbi request reprint Adeno-associated virus vector-mediated systemic delivery of IFN-beta combined with low-dose cyclophosphamide affects tumor regression in murine neuroblastoma models
    Christian J Streck
    Department of Surgery, St Jude Children s Research Hospital, Memphis, TN, USA
    Clin Cancer Res 11:6020-9. 2005
    ..We hypothesized that the antitumor activity of type I IFNs could be enhanced by chronic, low-dose systemic delivery and sought to test this in murine neuroblastoma models...
  28. ncbi request reprint Adeno-associated virus vector-mediated delivery of pigment epithelium-derived factor restricts neuroblastoma angiogenesis and growth
    Christian J Streck
    Department of Surgery, St Jude Children s Research Hospital and University of Tennessee Memphis Health Science Center, Memphis, TN 38105, USA
    J Pediatr Surg 40:236-43. 2005
    ..Pigment epithelium-derived factor was chosen for this study because, in addition to being a potent inhibitor of angiogenesis, it is capable of inducing neuronal differentiation...
  29. ncbi request reprint 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
    Chara A Kyriakou
    Department of Haematology, University College London, London, UK
    J Gene Med 8:253-64. 2006
    ..Efficient gene transfer to bone marrow derived mesenchymal stem cells (MSC) would provide an important opportunity to express potent anticancer agents in the tumour microenvironment because of their contribution to the tumour stroma...
  30. ncbi request reprint A new xenograft model of myeloma bone disease demonstrating the efficacy of human mesenchymal stem cells expressing osteoprotegerin by lentiviral gene transfer
    N Rabin
    Department of Haematology, University College London, London, UK
    Leukemia 21:2181-91. 2007
    ..Because of its predominantly medullary involvement and quantifiable parameters of bone disease, the KMS-12-BM xenogeneic model provides unique opportunities to test therapies targeted at the bone marrow microenvironment...
  31. ncbi request reprint Antiangiogenic gene therapy for cancer treatment
    Andrew M Davidoff
    Department of Surgery, St Jude Children s Research Hospital, 332 North Lauderdale, Memphis, TN 38105, USA
    Curr Hematol Rep 3:267-73. 2004
    ..This review summarizes the major alternatives within each of these categories and provides illustrative examples of their use in preclinical models...
  32. ncbi request reprint Restriction of neuroblastoma angiogenesis and growth by interferon-alpha/beta
    Christian J Streck
    Department of Surgery, St Jude Children s Research Hospital and the University of Tennessee Health Science Center, Memphis, TN 38105, USA
    Surgery 136:183-9. 2004
    ..We tested the hypothesis that the antiangiogenic activity of the type I interferons (IFNs), could affect tumor engraftment and growth in murine xenograft models of neuroblastoma...
  33. ncbi request reprint Interferon-mediated anti-angiogenic therapy for neuroblastoma
    Christian J Streck
    Department of Surgery, St Jude Children s Research Hospital, Memphis, Tennessee, USA
    Cancer Lett 228:163-70. 2005
    ..We have, therefore, explored the utility of a gene therapy-mediated approach for the delivery of interferon-beta and tested this approach, both alone and in combination with conventional chemotherapy, in murine models of neuroblastoma...
  34. ncbi request reprint Continuous delivery of human type I interferons (alpha/beta) has significant activity against acute myeloid leukemia cells in vitro and in a xenograft model
    Reuben Benjamin
    Department of Haematology, University College London, UK, and Division of Experimental Hematology, Department of Surgery, St Jude s Children s Research Hospital, Memphis, TN, USA
    Blood 109:1244-7. 2007
    ..These levels of IFN-beta are likely to be nontoxic in humans. Therefore, approaches capable of maintaining stable plasma levels of IFN-beta merit further clinical evaluation in patients with AML...
  35. ncbi request reprint Continuous delivery of IFN-beta promotes sustained maturation of intratumoral vasculature
    Paxton V Dickson
    Department of Surgery, St Jude Children s Research Hospital, 332 North Lauderdale, Memphis, TN 38105, USA
    Mol Cancer Res 5:531-42. 2007
    ..These results have significant implications for the planning of combination anticancer therapy...
  36. ncbi request reprint Delivery of antiangiogenic agents for cancer gene therapy
    Paxton V Dickson
    Department of Surgery, St Jude Children s Research Hospital, and the Department of Surgery, The University of Tennessee Memphis, Health Science Center, TN 38163, USA
    Technol Cancer Res Treat 4:331-41. 2005
    ..Here we review the major alternatives within each of these categories and provide illustrative examples of their use in preclinical models...