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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

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

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

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

NARROW

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

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

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

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.

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ASH Publications

American Society of Hematology

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

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

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

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.

Email alerts

ASH Publications

American Society of Hematology

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