Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A-Reference-Cited by-同舟云学术

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Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A

Published:2016-11-25 Issue:1 Volume:15 Page:110-121
ISSN:1538-7933
Container-title:Journal of Thrombosis and Haemostasis
language:en
Short-container-title:J Thromb Haemost

Author:

Nguyen G. N.¹,George L. A.¹²,Siner J. I.¹,Davidson R. J.¹,Zander C. B.³,Zheng X. L.³,Arruda V. R.¹²,Camire R. M.¹²,Sabatino D. E.¹²

Affiliation:

1. The Raymond G. Perelman Center for Cellular and Molecular Therapeutics; The Children's Hospital of Philadelphia; Philadelphia PA USA

2. Department of Pediatrics; Division of Hematology; Perelman School of Medicine; University of Pennsylvania; Philadelphia PA USA

3. Division of Laboratory Medicine; Department of Pathology; University of Alabama at Birmingham; Birmingham AL USA

Funder

Pfizer

National Hemophilia Foundation

National Institutes of Health

Publisher

Wiley

Subject

Hematology

Link

http://onlinelibrary.wiley.com/wol1/doi/10.1111/jth.13543/fullpdf

Reference40 articles.

1. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response;Manno;Nat Med,2006

2. Long-term safety and efficacy of factor IX gene therapy in hemophilia B;Nathwani;N Engl J Med,2014

3. Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors;Sabatino;Mol Ther,2011

4. Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs;Jiang;Blood,2006

5. Synthesis, processing, and secretion of recombinant human factor VIII expressed in mammalian cells;Kaufman;J Biol Chem,1988

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1. Pre-clinical evaluation of an enhanced-function factor VIII variant for durable hemophilia A gene therapy in male mice;Nature Communications;2024-08-21

2. Characterization of zebrafish coagulation cofactors Fviii and Fv mutants and modeling hemophilia A and factor V deficiency;Blood Coagulation & Fibrinolysis;2024-06-10

3. An adeno-associated virus variant enabling efficient ocular-directed gene delivery across species;Nature Communications;2024-05-06

4. AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life;Molecular Therapy - Methods & Clinical Development;2024-03

5. Functional Roles of Furin in Cardio-Cerebrovascular Diseases;ACS Pharmacology & Translational Science;2024-02-07

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