A Novel Cell-Sheet Technology That Achieves Durable Factor VIII Delivery in a Mouse Model of Hemophilia A
Author:
Publisher
Public Library of Science (PLoS)
Subject
Multidisciplinary
Reference28 articles.
1. Characterization of the human factor VIII gene;J Gitschier;Nature,1984
2. Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors;MK Chuah;Blood,2003
3. Helper-dependent adenoviral vectors mediate therapeutic factor VIII expression for several months with minimal accompanying toxicity in a canine model of severe hemophilia A;BD Brown;Blood,2004
4. Multiyear therapeutic benefit of AAV serotypes 2, 6 and 8 delivering factor VIII to hemophilia A mice and dogs;H Jiang;Blood,2006
5. Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors;DE Sabatino;Mol Ther,2011
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