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AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

The New England journal of medicine, 2017-12, Vol.377 (26), p.2519-2530

2017

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Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A

The New England journal of medicine, 2020-01, Vol.382 (1), p.29-40

2020

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Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A

Nature medicine, 2022-04, Vol.28 (4), p.789-797

2022

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Fc-fusion proteins and FcRn: structural insights for longer-lasting and more effective therapeutics

Critical reviews in biotechnology, 2015-06, Vol.35 (2), p.235-254

2015

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Activity of transgene-produced B-domain–deleted factor VIII in human plasma following AAV5 gene therapy

Blood, 2020-11, Vol.136 (22), p.2524-2534

2020

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Phase 3 Study of Recombinant Factor IX Fc Fusion Protein in Hemophilia B

The New England journal of medicine, 2013-12, Vol.369 (24), p.2313-2323

2013

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Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients

Blood, 2012-03, Vol.119 (13), p.3031-3037

2012

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Uncertainty in an era of transformative therapy for haemophilia: Addressing the unknowns

Haemophilia : the official journal of the World Federation of Hemophilia, 2021-02, Vol.27 (S3), p.103-113

Pierce, Glenn F.

2021

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Von Willebrand Disease: Gaining a global perspective

Haemophilia : the official journal of the World Federation of Hemophilia, 2023-07, Vol.29 (4), p.1104-1112

2023

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World Federation of Hemophilia Gene Therapy Registry

Haemophilia : the official journal of the World Federation of Hemophilia, 2020-07, Vol.26 (4), p.563-564

2020

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Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs

Blood, 2012-03, Vol.119 (13), p.3024-3030

2012

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Recombinant factor VIII Fc (rFVIIIFc) fusion protein reduces immunogenicity and induces tolerance in hemophilia A mice

Cellular immunology, 2016-03, Vol.301, p.30-39

2016

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Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy

Blood, 2006-11, Vol.108 (10), p.3321-3328

2006

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Gene Therapy for Hemophilia: Are Expectations Matching Reality?

Molecular therapy, 2020-10, Vol.28 (10), p.2097-2098

Pierce, Glenn F.

2020

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Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver

Blood, 2007-10, Vol.110 (7), p.2334-2341

2007

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Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A

The New England journal of medicine, 2020-01, Vol.382 (1), p.29

2020

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Switching to recombinant factor IX Fc fusion protein prophylaxis results in fewer infusions, decreased factor IX consumption and lower bleeding rates

British journal of haematology, 2015-01, Vol.168 (1), p.113-123

2015

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Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice

Blood, 2006-03, Vol.107 (5), p.1810-1817

2006

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Recombinant factor IX Fc fusion protein in children with haemophilia B (Kids B-LONG): results from a multicentre, non-randomised phase 3 study

The Lancet. Haematology, 2017-02, Vol.4 (2), p.e75-e82

2017

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Mutations on the External Surfaces of Adeno-Associated Virus Type 2 Capsids That Affect Transduction and Neutralization

Journal of Virology, 2006-01, Vol.80 (2), p.821-834

2006

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The COVID‐19 pandemic: New global challenges for the haemophilia community

Haemophilia : the official journal of the World Federation of Hemophilia, 2020-05, Vol.26 (3), p.371-372

2020

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Towards achieving a haemophilia‐free mind

Haemophilia : the official journal of the World Federation of Hemophilia, 2023-07, Vol.29 (4), p.951-953

2023

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Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B

Blood, 2010-06, Vol.115 (23), p.4678-4688

2010

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AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A

Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A

Fc-fusion proteins and FcRn: structural insights for longer-lasting and more effective therapeutics

Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A

Activity of transgene-produced B-domain–deleted factor VIII in human plasma following AAV5 gene therapy

Phase 3 Study of Recombinant Factor IX Fc Fusion Protein in Hemophilia B

Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients

Uncertainty in an era of transformative therapy for haemophilia: Addressing the unknowns

Von Willebrand Disease: Gaining a global perspective

World Federation of Hemophilia Gene Therapy Registry

Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs

Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients

Recombinant factor VIII Fc (rFVIIIFc) fusion protein reduces immunogenicity and induces tolerance in hemophilia A mice

Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy

Gene Therapy for Hemophilia: Are Expectations Matching Reality?

Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver

Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A

Switching to recombinant factor IX Fc fusion protein prophylaxis results in fewer infusions, decreased factor IX consumption and lower bleeding rates

Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice

Recombinant factor IX Fc fusion protein in children with haemophilia B (Kids B-LONG): results from a multicentre, non-randomised phase 3 study

Mutations on the External Surfaces of Adeno-Associated Virus Type 2 Capsids That Affect Transduction and Neutralization

The COVID‐19 pandemic: New global challenges for the haemophilia community

Towards achieving a haemophilia‐free mind

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B

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