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Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy

31 Dec 2018

Abstract excerpt

Hemophilia A (HA) and hemophilia B (HB) are X-linked bleeding disorders due to inheritable deficiencies in either coagulation factor VIII (FVIII) or factor IX (FIX), respectively. Recently, gene therapy clinical trials with adeno-associated virus (AAV) vectors and protein-engineered transgenes, B-domain deleted (BDD) FVIII and FIX-Padua, have reported near-phenotypic cures in subjects with HA and HB,...

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Share to Blood Coagulation and Thrombosis Mechanisms
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PMID: 30705923
DOI: 10.1016/j.omtm.2018.12.007

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