Adeno-associated virus-based gene therapy for hemophilia–addressing the gaps

Adeno-associated virus-based gene therapy for hemophilia–addressing the gaps

Adeno-associated virus-based gene therapy for hemophilia has emerged as a revolutionary treatment option, offering potential correction of clotting factor deficiency through a single intravenous infusion of functional genes directed to hepatocytes. With 3 gene therapies recently approved, this appro...

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Bibliographic Details
Main Authors: Wolfgang Miesbach, Paul Batty, Pratima Chowdary, Sylvia Fong, Radoslaw Kaczmarek, Frank W.G. Leebeek, Brian Long, Johnny Mahlangu, Mike Makris, Glenn F. Pierce, Steven W. Pipe, Alok Srivastava, Jan Voorberg, Flora Peyvandi
Format: Article
Language:English
Published: Elsevier 2025-01-01
Series:Research and Practice in Thrombosis and Haemostasis
Subjects:
gene therapy
hemophilia
methods
safety
standardization
Online Access:http://www.sciencedirect.com/science/article/pii/S2475037924003686
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