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Journal Articles

Enhancing FVIII Expression By Modifying B-Domain Sequence for Hemophilia A Gene Therapy

Doyoung Lee, PhD, Jenny McIntosh, PhD, Allison Dane, PhD, Cecilia Rosales, PhD, Anne Riddell, Edward Tuddenham, MD, Amit C Nathwani, MD PhD

Blood (2018) 132 (Supplement 1): 3484.

https://doi.org/10.1182/blood-2018-99-113830

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

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

Blood (2013) 122 (21): 717.

https://doi.org/10.1182/blood.V122.21.717.717

Published: 2013

Journal Articles

Assessing the Potential of Perinatal Gene Transfer Using Congenital Factor VII Deficiency as a Model System

Christopher J Binny, Jenny H McIntosh, Edward Tuddenham, MD, Simon Waddington, Suzanne Buckley, Adrian Thrasher, John T. Gray, PhD, Amit Nathwani, MD, PhD, Alice F Tarantal, PhD, Andrew Davidoff

Blood (2010) 116 (21): 247.

https://doi.org/10.1182/blood.V116.21.247.247

Published: 2010

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

Blood (2010) 116 (21): 248.

https://doi.org/10.1182/blood.V116.21.248.248

Published: 2010

Journal Articles

Stable High Level Coagulation Factor VIII Expression In Vivo Following Gene Transfer Using a Novel Expression Cassette Encoding a More Potent FVIII Variant

Jenny McIntosh, Peter J Lenting, Edward Tuddenham, MD, Motunrayo Sotannde, Simon Waddington, John T. Gray, PhD, Andrew Davidoff, Amit Nathwani, MD, PhD

Blood (2010) 116 (21): 250.

https://doi.org/10.1182/blood.V116.21.250.250

Published: 2010

Journal Articles

Mutations in the VKORC1 Gene Cause Warfarin Resistance, Warfarin Sensitivity and Combined Deficiency of Vitamin K Dependent Coagulation Factors.

Johannes Oldenburg, MD, PhD, Simone Rost, Andreas Fregin, PhD, Christof Geisen, Vytautas Ivaskevicius, MD, Erhard Seifried, MD, Inge Scharrer, MD, Maximilian Heistinger, MD, Edward Tuddenham, MD, Clemens Muller-Reible, PhD, Barbara Zieger, MD

Blood (2004) 104 (11): 277.

https://doi.org/10.1182/blood.V104.11.277.277

Published: 2004

NARROW

Enhancing FVIII Expression By Modifying B-Domain Sequence for Hemophilia A Gene Therapy

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

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

Assessing the Potential of Perinatal Gene Transfer Using Congenital Factor VII Deficiency as a Model System

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

Stable High Level Coagulation Factor VIII Expression In Vivo Following Gene Transfer Using a Novel Expression Cassette Encoding a More Potent FVIII Variant

Mutations in the VKORC1 Gene Cause Warfarin Resistance, Warfarin Sensitivity and Combined Deficiency of Vitamin K Dependent Coagulation Factors.

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ASH Publications

American Society of Hematology

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ASH Publications

American Society of Hematology

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