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

Blood, 2012-01, Vol.119 (3), p.666-672

2012

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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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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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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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Novel approach to genetic analysis and results in 3000 hemophilia patients enrolled in the My Life, Our Future initiative

Blood advances, 2017-05, Vol.1 (13), p.824-834

2017

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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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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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First conditional marketing authorization approval in the European Union for hemophilia “A” gene therapy

Molecular therapy, 2022-11, Vol.30 (11), p.3335-3336

2022

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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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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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Two gene therapies for hemophilia available: Now what?

Molecular therapy, 2023-04, Vol.31 (4), p.919-920

2023

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Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs

Blood, 2006-07, Vol.108 (1), p.107-115

2006

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Deciphering conundrums of adeno-associated virus liver-directed gene therapy: focus on hemophilia

Journal of thrombosis and haemostasis, 2024-05, Vol.22 (5), p.1263-1289

2024

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Reimbursing the value of gene therapy care in an era of uncertainty

Haemophilia : the official journal of the World Federation of Hemophilia, 2021-01, Vol.27 (1), p.12-18

2021

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Transient expression of factor VIII and a chronic high-fat diet induces ER stress and late hepatocyte oncogenesis

Molecular therapy, 2022-12, Vol.30 (12), p.3510-3512

2022

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Impact of humanitarian aid linked prophylaxis in Côte d'Ivoire (Ivory Coast)

Haemophilia : the official journal of the World Federation of Hemophilia, 2021-01, Vol.27 (1), p.9-11

2021

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Eliminating Panglossian thinking in development of AAV therapeutics

Molecular therapy, 2021-12, Vol.29 (12), p.3325-3327

2021

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Treatment of nonhealing diabetic foot ulcers with a platelet-derived growth factor gene-activated matrix (GAM501): Results of a Phase 1/2 trial

Wound repair and regeneration, 2009-11, Vol.17 (6), p.772-779

2009

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

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

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

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

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

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?

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

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

Novel approach to genetic analysis and results in 3000 hemophilia patients enrolled in the My Life, Our Future initiative

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

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

First conditional marketing authorization approval in the European Union for hemophilia “A” gene therapy

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

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

Two gene therapies for hemophilia available: Now what?

Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs

Deciphering conundrums of adeno-associated virus liver-directed gene therapy: focus on hemophilia

Reimbursing the value of gene therapy care in an era of uncertainty

Transient expression of factor VIII and a chronic high-fat diet induces ER stress and late hepatocyte oncogenesis

Impact of humanitarian aid linked prophylaxis in Côte d'Ivoire (Ivory Coast)

Eliminating Panglossian thinking in development of AAV therapeutics

Treatment of nonhealing diabetic foot ulcers with a platelet-derived growth factor gene-activated matrix (GAM501): Results of a Phase 1/2 trial

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