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Journal Articles

Long-Term Persistence of Antibodies to Adeno-Associated Viral Vectors Following Gene Therapy with scAAV8-LP1-Fixco

Jenny H. McIntosh, Saira Mohamed, Lydia Ireland, Christopher L Morton, Ulrike M. Reiss, Pratima Chowdary, Edward G. D. Tuddenham, Andrew M. Davidoff, Amit C. Nathwani

Blood (2023) 142 (Supplement 1): 2255.

https://doi.org/10.1182/blood-2023-188383

Published: 2023

Journal Articles

Stable Therapeutic Transgenic FIX Levels for More Than 10 Years in Subjects with Severe Hemophilia B Who Received scAAV2/8-LP1-Hfixco Adeno-Associated Virus Gene Therapy

Blood (2023) 142 (Supplement 1): 1056.

https://doi.org/10.1182/blood-2023-186891

Published: 2023

Journal Articles

GO-8: Stable Expression of Factor VIII over 5 Years Following Adeno-Associated Gene Transfer in Subjects with Hemophilia a Using a Novel Human Factor VIII Variant

Blood (2023) 142 (Supplement 1): 3624.

https://doi.org/10.1182/blood-2023-180803

Published: 2023

Journal Articles

The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study from the United States perspective

Nancy S. Bolous, Yichen Chen, Huiqi Wang, Andrew M. Davidoff, Meenakshi Devidas, Timothy W. Jacobs, Michael M. Meagher, Amit C. Nathwani, Ellis J. Neufeld, Bryan A. Piras, Carlos Rodriguez-Galindo, Ulrike M. Reiss, Nickhill Bhakta

Blood (2021) 138 (18): 1677–1690.

https://doi.org/10.1182/blood.2021010864

Published: 2021

Includes: Supplemental data

Journal Articles

Preclinical Evaluation of an Engineered AAV Capsid in Non-Human Primates for the Treatment of Haemophilia B

Allison Dane, PhD, Jenny McIntosh, Doyoung Lee, PhD, Rose Sheridan, Mark Maginn, Romuald Corbau, PhD, Andrew M. Davidoff, MD, Amit C Nathwani, MD PhD

Blood (2018) 132 (Supplement 1): 2197.

https://doi.org/10.1182/blood-2018-99-117969

Published: 2018

Journal Articles

Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer

Amit C Nathwani, MD PhD, Ulrike Reiss, MD, Edward Tuddenham, MD, Pratima Chowdary, MD, Jenny McIntosh, PhD, Anne Riddell, Jun Pie, Johnny N Mahlangu, MD, Michael Recht, MD PhD, Yu-Min Shen, MD, Kathleen G. Halka, MD, Michael M Meagher, PhD, Arthur W. Nienhuis, MD, Andrew M. Davidoff, MD, Sarah Mangles, Chris L Morton, Zhou Junfang, Vlad Calin Radulescu, MD

Blood (2018) 132 (Supplement 1): 491.

https://doi.org/10.1182/blood-2018-99-118334

Published: 2018

Journal Articles

GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant

Amit C Nathwani, MD PhD, Edward Tuddenham, MD, Pratima Chowdary, MD, Jenny McIntosh, PhD, Doyoung Lee, PhD, Cecilia Rosales, PhD, Mark Phillips, Jun Pie, Zhou Junfang, Michael M Meagher, PhD, Ulrike Reiss, MD, Andrew M. Davidoff, MD, Chris L Morton, Anne Riddell

Blood (2018) 132 (Supplement 1): 489.

https://doi.org/10.1182/blood-2018-99-118256

Published: 2018

Journal Articles

A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B

Pratima Chowdary, MD, Susie Shapiro, MD, Andrew M. Davidoff, MD, Ulrike Reiss, MD, Rebecca Alade, PhD, Geoff Brooks, BSc, Allison Dane, PhD, Jenny McIntosh, PhD, Gerard Short, MBBS, Edward Tuddenham, MD, Amit C Nathwani, MD PhD

Blood (2018) 132 (Supplement 1): 631.

https://doi.org/10.1182/blood-2018-99-118050

Published: 2018

Journal Articles

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

Blood (2013) 121 (17): 3335–3344.

https://doi.org/10.1182/blood-2012-10-462200

Published: 2013

Includes: Supplemental data

Journal Articles

AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage

Christopher Binny, Jenny McIntosh, Marco Della Peruta, Hanna Kymalainen, Edward G. D. Tuddenham, Suzanne M. K. Buckley, Simon N. Waddington, John H. McVey, Yunyu Spence, Christopher L. Morton, Adrian J. Thrasher, John T. Gray, Francis J. Castellino, Alice F. Tarantal, Andrew M. Davidoff, Amit C. Nathwani

Blood (2012) 119 (4): 957–966.

https://doi.org/10.1182/blood-2011-09-377630

Published: 2012

Includes: Supplemental data

Journal Articles

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, John T. Gray, Jenny McIntosh, Catherine Y. C. Ng, Junfang Zhou, Yunyu Spence, Melanie Cochrane, Elaine Gray, Edward G. D. Tuddenham, Andrew M. Davidoff

Blood (2006) 109 (4): 1414–1421.

https://doi.org/10.1182/blood-2006-03-010181

Published: 2006

Journal Articles

Continuous delivery of human type I interferons (α/β) has significant activity against acute myeloid leukemia cells in vitro and in a xenograft model

Reuben Benjamin, Asim Khwaja, Nalini Singh, Jenny McIntosh, Anthony Meager, Meenu Wadhwa, Christian Streck, Catherine Ng, Andrew M. Davidoff, Amit C. Nathwani

Blood (2006) 109 (3): 1244–1247.

https://doi.org/10.1182/blood-2006-02-002915

Published: 2006

Journal Articles

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, John T. Gray, Catherine Y. C. Ng, Junfang Zhou, Yunyu Spence, Simon N. Waddington, Edward G. D. Tuddenham, Geoffrey Kemball-Cook, Jenny McIntosh, Mariette Boon-Spijker, Koen Mertens, Andrew M. Davidoff

Blood (2006) 107 (7): 2653–2661.

https://doi.org/10.1182/blood-2005-10-4035

Published: 2006

Includes: Supplemental data

Journal Articles

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.

John T. Gray, PhD, Andrew M. Davidoff, MD, Amit C. Nathwani, MD, PhD

Blood (2004) 104 (11): 3181.

https://doi.org/10.1182/blood.V104.11.3181.3181

Published: 2004

Journal Articles

Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway

Andrew M. Davidoff, Catherine Y. C. Ng, Junfang Zhou, Yunyu Spence, Amit C. Nathwani

Blood (2003) 102 (2): 480–488.

https://doi.org/10.1182/blood-2002-09-2889

Published: 2003

Journal Articles

Enforced expression of tissue inhibitor of matrix metalloproteinase-3 affects functional capillary morphogenesis and inhibits tumor growth in a murine tumor model

William W. Spurbeck, Catherine Y. C. Ng, Ted S. Strom, Elio F. Vanin, Andrew M. Davidoff

Blood (2002) 100 (9): 3361–3368.

https://doi.org/10.1182/blood.V100.9.3361

Published: 2002

Journal Articles

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, Andrew M. Davidoff, Hideki Hanawa, Yunyu Hu, Fredric A. Hoffer, Alexander Nikanorov, Clive Slaughter, Catherine Y. C. Ng, Junfang Zhou, Jay N. Lozier, Timothy D. Mandrell, Elio F. Vanin, Arthur W. Nienhuis

Blood (2002) 100 (5): 1662–1669.

https://doi.org/10.1182/blood-2002-02-0589

Published: 2002

NARROW

Long-Term Persistence of Antibodies to Adeno-Associated Viral Vectors Following Gene Therapy with scAAV8-LP1-Fixco

Stable Therapeutic Transgenic FIX Levels for More Than 10 Years in Subjects with Severe Hemophilia B Who Received scAAV2/8-LP1-Hfixco Adeno-Associated Virus Gene Therapy

GO-8: Stable Expression of Factor VIII over 5 Years Following Adeno-Associated Gene Transfer in Subjects with Hemophilia a Using a Novel Human Factor VIII Variant

The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study from the United States perspective

Preclinical Evaluation of an Engineered AAV Capsid in Non-Human Primates for the Treatment of Haemophilia B

Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer

GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant

A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage

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

Continuous delivery of human type I interferons (α/β) has significant activity against acute myeloid leukemia cells in vitro and in a xenograft model

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

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.

Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway

Enforced expression of tissue inhibitor of matrix metalloproteinase-3 affects functional capillary morphogenesis and inhibits tumor growth in a murine tumor model

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

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American Society of Hematology

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American Society of Hematology

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