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Journal Articles

Nfix is a novel regulator of murine hematopoietic stem and progenitor cell survival

Per Holmfeldt, Jennifer Pardieck, Anjelica C. Saulsberry, Satish Kumar Nandakumar, David Finkelstein, John T. Gray, Derek A. Persons, Shannon McKinney-Freeman

Blood (2013) 122 (17): 2987–2996.

https://doi.org/10.1182/blood-2013-04-493973

Published: 2013

Includes: Supplemental data

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

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

Blood (2012) 120 (21): 752.

https://doi.org/10.1182/blood.V120.21.752.752

Published: 2012

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

Transduction of Human CD34+ Derived NSG Repopulating Cells with An Insulated SIN Lentiviral Vector for SCID-X1 From a Stable Producer Cell Line At Clinical Scale

Michael R. Greene, MD, Timothy Lockey, PhD, John T. Gray, PhD, Paul W. Eldridge, PhD, Yoon-Sang Kim, MD, PhD, Perdeep K. Mehta, PhD, Brian P. Sorrentino, MD

Blood (2011) 118 (21): 670.

https://doi.org/10.1182/blood.V118.21.670.670

Published: 2011

Journal Articles

Adeno-Associated Viral Vector Mediated Gene Transfer for Hemophilia B

Blood (2011) 118 (21): 5.

https://doi.org/10.1182/blood.V118.21.5.5

Published: 2011

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

A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells

Sheng Zhou, Disha Mody, Suk See DeRavin, Julia Hauer, Taihe Lu, Zhijun Ma, Salima Hacein-Bey Abina, John T. Gray, Michael R. Greene, Marina Cavazzana-Calvo, Harry L. Malech, Brian P. Sorrentino

Blood (2010) 116 (6): 900–908.

https://doi.org/10.1182/blood-2009-10-250209

Published: 2010

Includes: Supplemental data

Journal Articles

Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection

Robert E. Throm, Annastasia A. Ouma, Sheng Zhou, Anantharaman Chandrasekaran, Timothy Lockey, Michael Greene, Suk See De Ravin, Morvarid Moayeri, Harry L. Malech, Brian P. Sorrentino, John T. Gray

Blood (2009) 113 (21): 5104–5110.

https://doi.org/10.1182/blood-2008-11-191049

Published: 2009

Journal Articles

An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation

Byoung Y. Ryu, Marguerite V. Evans-Galea, John T. Gray, David M. Bodine, Derek A. Persons, Arthur W. Nienhuis

Blood (2008) 111 (4): 1866–1875.

https://doi.org/10.1182/blood-2007-04-085506

Published: 2008

Includes: Supplemental data

Journal Articles

The Effect of a Chromatin Insulator (5′cHS4) on Enhanced Expression of the LMO2 Proto-Oncogene by an Oncoretroviral Long Terminal Repeat.

Byoung Y. Ryu, PhD, John T. Gray, PhD, David M. Bodine, PhD, Arthur W. Nienhuis, MD

Blood (2006) 108 (11): 3253.

https://doi.org/10.1182/blood.V108.11.3253.3253

Published: 2006

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

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

NARROW

Nfix is a novel regulator of murine hematopoietic stem and progenitor cell survival

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

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

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

Transduction of Human CD34+ Derived NSG Repopulating Cells with An Insulated SIN Lentiviral Vector for SCID-X1 From a Stable Producer Cell Line At Clinical Scale

Adeno-Associated Viral Vector Mediated Gene Transfer for Hemophilia B

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

A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells

Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection

An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation

The Effect of a Chromatin Insulator (5′cHS4) on Enhanced Expression of the LMO2 Proto-Oncogene by an Oncoretroviral Long Terminal Repeat.

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

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.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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