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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

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

Blood (2023) 142 (Supplement 1): 3624.

https://doi.org/10.1182/blood-2023-180803

Published: 2023

Journal Articles

Long-Term Persistence of Antibodies to Adeno-Associated Viral Vectors Following Gene Therapy with scAAV8-LP1-Fixco

Jenny H. McIntosh, Saira Mohamed, Lydia Ireland, Christopher L Morton, Ulrike M. Reiss, Pratima Chowdary, Edward G. D. Tuddenham, Andrew M. Davidoff, Amit C. Nathwani

Blood (2023) 142 (Supplement 1): 2255.

https://doi.org/10.1182/blood-2023-188383

Published: 2023

Journal Articles

Solution structure of the major factor VIII binding region on von Willebrand factor

Nuha Shiltagh, John Kirkpatrick, Lisa D. Cabrita, Tom A. J. McKinnon, Konstantinos Thalassinos, Edward G. D. Tuddenham, D. Flemming Hansen

Blood (2014) 123 (26): 4143–4151.

https://doi.org/10.1182/blood-2013-07-517086

Published: 2014

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

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

Codon optimization of human factor VIII cDNAs leads to high-level expression

Natalie J. Ward, Suzanne M. K. Buckley, Simon N. Waddington, Thierry VandenDriessche, Marinee K. L. Chuah, Amit C. Nathwani, Jenny McIntosh, Edward G. D. Tuddenham, Christine Kinnon, Adrian J. Thrasher, John H. McVey

Blood (2011) 117 (3): 798–807.

https://doi.org/10.1182/blood-2010-05-282707

Published: 2011

Includes: Supplemental data

Journal Articles

Killing 2 birds with 1 stone

Edward G. D. Tuddenham

Blood (2008) 112 (7): 2595.

https://doi.org/10.1182/blood-2008-07-163030

Published: 2008

Journal Articles

Autosomal dominant erythrocytosis and pulmonary arterial hypertension associated with an activating HIF2 α mutation

Daniel P. Gale, Sarah K. Harten, Cecil D. L. Reid, Edward G. D. Tuddenham, Patrick H. Maxwell

Blood (2008) 112 (3): 919–921.

https://doi.org/10.1182/blood-2008-04-153718

Published: 2008

Journal Articles

Genotype-phenotype correlation in combined deficiency of factor V and factor VIII

Bin Zhang, Marta Spreafico, Chunlei Zheng, Angela Yang, Petra Platzer, Michael U. Callaghan, Zekai Avci, Namik Ozbek, Johnny Mahlangu, Tabitha Haw, Randal J. Kaufman, Kandice Marchant, Edward G. D. Tuddenham, Uri Seligsohn, Flora Peyvandi, David Ginsburg

Blood (2008) 111 (12): 5592–5600.

https://doi.org/10.1182/blood-2007-10-113951

Published: 2008

Includes: Supplemental data

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

Combined deficiency of factor V and factor VIII is due to mutations in either LMAN1 or MCFD2

Bin Zhang, Beth McGee, Jennifer S. Yamaoka, Hugo Guglielmone, Katharine A. Downes, Salvador Minoldo, Gustavo Jarchum, Flora Peyvandi, Norma B. de Bosch, Arlette Ruiz-Saez, Bernard Chatelain, Marian Olpinski, Paula Bockenstedt, Wolfgang Sperl, Randal J. Kaufman, William C. Nichols, Edward G. D. Tuddenham, David Ginsburg

Blood (2006) 107 (5): 1903–1907.

https://doi.org/10.1182/blood-2005-09-3620

Published: 2006

Journal Articles

Factor VIII: purer is not necessarily better

Edward G. D. Tuddenham

Blood (2006) 107 (1): 4–5.

https://doi.org/10.1182/blood-2005-10-4086

Published: 2006

Journal Articles

A novel missense mutation in ABCA1 results in altered protein trafficking and reduced phosphatidylserine translocation in a patient with Scott syndrome

Christiane Albrecht, John H. McVey, James I. Elliott, Alessandro Sardini, Ildiko Kasza, Andrew D. Mumford, Rossi P. Naoumova, Edward G. D. Tuddenham, Katalin Szabo, Christopher F. Higgins

Blood (2005) 106 (2): 542–549.

https://doi.org/10.1182/blood-2004-05-2056

Published: 2005

Journal Articles

Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy

Blood (2004) 104 (9): 2714–2721.

https://doi.org/10.1182/blood-2004-02-0627

Published: 2004

Journal Articles

Functional characterization of factor V-Ile359Thr: a novel mutation associated with thrombosis

Mårten Steen, Eva A. Norstrøm, Ann-Louise Tholander, Paula H. B. Bolton-Maggs, Andrew Mumford, John H. McVey, Edward G. D. Tuddenham, Björn Dahlbäck

Blood (2004) 103 (9): 3381–3387.

https://doi.org/10.1182/blood-2003-06-2092

Published: 2004

NARROW

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

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

Long-Term Persistence of Antibodies to Adeno-Associated Viral Vectors Following Gene Therapy with scAAV8-LP1-Fixco

Solution structure of the major factor VIII binding region on von Willebrand factor

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

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

Codon optimization of human factor VIII cDNAs leads to high-level expression

Killing 2 birds with 1 stone

Autosomal dominant erythrocytosis and pulmonary arterial hypertension associated with an activating HIF2 α mutation

Genotype-phenotype correlation in combined deficiency of factor V and factor VIII

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

Combined deficiency of factor V and factor VIII is due to mutations in either LMAN1 or MCFD2

Factor VIII: purer is not necessarily better

A novel missense mutation in ABCA1 results in altered protein trafficking and reduced phosphatidylserine translocation in a patient with Scott syndrome

Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy

Functional characterization of factor V-Ile359Thr: a novel mutation associated with thrombosis

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American Society of Hematology

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American Society of Hematology

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