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Journal Articles

Cellular Immune Responses To Vector In a Gene Therapy Trial For Hemophilia B Using An AAV8 Self-Complementary Factor IX Vector

Etiena Basner-Tschakarjan, MD PhD, Federico Mingozzi, PhD, Yifeng Chen, MD, Amit Nathwani, MD, PhD, Edward Tuddenham, MD, Anne Riddell, BSc, Catherine Ng, MS, Pradip Rustagi, MD, Bertil Glader, MD, PhD, Mark Kay, MD, PhD, Ulrike M. Reiss, MD, Arthur W. Nienhuis, MD, Andrew Davidoff, MD, Katherine A. High, MD
Journal: Blood
Blood (2013) 122 (21): 717.
https://doi.org/10.1182/blood.V122.21.717.717
Published: 2013
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

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

Amit Nathwani, MD, PhD, Edward Tuddenham, MD, Cecilia Rosales, Jenny McIntosh, Anne Riddell, Pradip Rustagi, Bertil Glader, Mark Kay, MD, PhD, James Allay, PhD, John Coleman, Susan Sleep, Katherine A. High, MD, Federico Mingozzi, PhD., John T. Gray, PhD, Ulrike M. Reiss, MD, Arthur W. Nienhuis, MD, Andrew Davidoff
Journal: Blood
Blood (2010) 116 (21): 248.
https://doi.org/10.1182/blood.V116.21.248.248
Published: 2010
Journal Articles

Human Immune Responses to AAV-2 Capsid May Limit Duration of Expression in Liver-Directed Gene Transfer in Humans with Hemophilia B.

Katherine High, MD, Michael Tigges, PhD, Catherine Manno, MD, Denise Sabatino, PhD, Valder Arruda, MD, Roland Herzog, PhD, Pradip Rustagi, MD, John Rasko, MD, Jurg Sommer, PhD, Kristen Jaworski, RN, Margaret Ragni, MD, Bertil Glader, MD, Ruth Lessard, Alvin Luk, PhD, Linda Couto, PhD, Haiyan Jiang, PhD, Glenn Pierce, MD, Mark Kay, MD
Journal: Blood
Blood (2004) 104 (11): 413.
https://doi.org/10.1182/blood.V104.11.413.413
Published: 2004