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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

Ulrike M. Reiss, Andrew M. Davidoff, Edward G. D. Tuddenham, Pratima Chowdary, Jenny H. McIntosh, Anne Riddell, Arnulfo Pie, Paul Batty, Joanna C. M. Calvert, Sarah Mangles, Johnny Mahlangu, Kristina M. Haley, Michael Recht, Yu-Min Shen, Kathleen G. Halka, Junfang Zhou, Guolian Kang, Victoria Okhomina, Christopher L Morton, Amit C. Nathwani
Journal: Blood
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

Pratima Chowdary, Ulrike M. Reiss, Edward G. D. Tuddenham, Paul Batty, Jenny H. McIntosh, Vlad Calin Radulescu, Eugenia Chang, Michael A. Laffan, Anne Riddell, Joanna C. M. Calvert, Arnulfo Pie, Rita Peralta, Upuli Pabakumari Dissanayake, Kirollos Kamel, Bharath Ram Sreedhar, Junfang Zhou, Guolian Kang, Yunus Olufadi, Michael M Meagher, Christopher L Morton, Alison J. Evans, Andrew M. Davidoff, Amit C. Nathwani
Journal: Blood
Blood (2023) 142 (Supplement 1): 3624.
https://doi.org/10.1182/blood-2023-180803
Published: 2023
Journal Articles

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

Jenny McIntosh, Peter J. Lenting, Cecilia Rosales, Doyoung Lee, Samira Rabbanian, Deepak Raj, Nishil Patel, Edward G. D. Tuddenham, Olivier D. Christophe, John H. McVey, Simon Waddington, Arthur W. Nienhuis, John T. Gray, Paolo Fagone, Federico Mingozzi, Shang-Zhen Zhou, Katherine A. High, Maria Cancio, Catherine Y. C. Ng, Junfang Zhou, Christopher L. Morton, Andrew M. Davidoff, Amit C. Nathwani
Journal: Blood
Blood (2013) 121 (17): 3335–3344.
https://doi.org/10.1182/blood-2012-10-462200
Published: 2013
Includes: Supplemental data
Journal Articles

Stable Factor IX Activity Following AAV-Mediated Gene Transfer in Patients with Severe Hemophilia B

Andrew Davidoff, MD, Edward GD Tuddenham, MBBS, FRCP, FRCPath, PhD, Savita Rangarajan, MBBS, FRCP, FRCPath, Cecilia Rosales, PhD, Jenny McIntosh, PhD, Pratima Chowdary, MBBS, FRCP, FRCPath, Anne Riddell, BSc, Bertil Glader, MD, PhD, Pradip Rustagi, MD, Catherine Ng, MS, Mark Kay, MD, PhD, Junfang Zhou, MD, Yunyu Spence, PhD, Christopher Morton, B.S., James Allay, PhD, Susan Sleep, PhD, Etiena Basner-Tschakarjan, MD, Federico Mingozzi, PhD., Katherine A. High, MD, John T. Gray, PhD, Ulrike M. Reiss, MD, Arthur W. Nienhuis, MD, Amit C Nathwani, MBChB, FRCP, FRCPath, PhD
Journal: Blood
Blood (2012) 120 (21): 752.
https://doi.org/10.1182/blood.V120.21.752.752
Published: 2012
Journal Articles

Adeno-Associated Viral Vector Mediated Gene Transfer for Hemophilia B

Amit C Nathwani, MBChB, FRCP, FRCPath, PhD, Edward GD Tuddenham, MBBS, FRCP, FRCPath, PhD, Savita Rangarajan, MBBS, FRCP, FRCPath, Cecilia Rosales, PhD, Jenny H McIntosh, PhD, David C Linch, MD FMedSci, Pratima Chowdary, MBBS, FRCP, FRCPath, Anja Griffioen, Anne Riddell, BSc, Jun Pie, BSN, Chris Harrington, BSN, James O’Beirne, MBBS, FRCP, MD EDIC, Keith Smith, BSc, MSc, C.Biol, John Pasi, MBChB, PhD, FRCP, FRCPath, Bertil Glader, MD PhD, Pradip Rustagi, MD, Catherine Ng, MS, Mark Kay, MD, PhD, Junfang Zhou, MD, Yunyu Spence, PhD, Christopher Morton, BS, James Allay, PhD, John Coleman, Susan Sleep, PhD, John M Cunningham, MD, Etiena Basner-Tschakarjan, MD, Federico Mingozzi, PhD, Katherine A. High, MD, John T. Gray, PhD, Ulrike M. Reiss, MD, Arthur W. Nienhuis, MD, Andrew Davidoff, MD
Journal: Blood
Blood (2011) 118 (21): 5.
https://doi.org/10.1182/blood.V118.21.5.5
Published: 2011
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
Journal: Blood
Blood (2006) 109 (4): 1414–1421.
https://doi.org/10.1182/blood-2006-03-010181
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
Journal: Blood
Blood (2006) 107 (7): 2653–2661.
https://doi.org/10.1182/blood-2005-10-4035
Published: 2006
Includes: Supplemental data
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
Journal: Blood
Blood (2003) 102 (2): 480–488.
https://doi.org/10.1182/blood-2002-09-2889
Published: 2003
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
Journal: Blood
Blood (2002) 100 (5): 1662–1669.
https://doi.org/10.1182/blood-2002-02-0589
Published: 2002