Gene-based therapies for hemophilia

Gene therapy is a transformative approach to treating genetic disorders in order to improve disease outcomes for patients. Hemophilia A and B are inherited genetic disorders caused by mutations in the FVIII and FIX genes, respectively. Traditional treatments for hemophilia have included intravenous...

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Bibliographic Details
Main Authors: Melissa F. Glasner, Steven Pipe, Wolfgang Miesbach
Format: Article
Language:English
Published: Elsevier 2025-05-01
Series:Research and Practice in Thrombosis and Haemostasis
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Online Access:http://www.sciencedirect.com/science/article/pii/S2475037925001943
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