Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery

Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery

Clinical data support the feasibility and safety of adeno-associated viral (AAV) vectors in gene therapy applications. Despite several clinical trials of AAV-based gene transfer for hemophilia B, a unique set of obstacles impede the development of a similar approach for hemophilia A. These include (...

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Bibliographic Details
Main Authors: Brown, Harrison C, Wright, J Fraser, Zhou, Shangzhen, Lytle, Allison M, Shields, Jordan E, Spencer, H Trent, Doering, Christopher B
Format: Online
Language:English
Published: Nature Publishing Group 2014
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4362354/

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https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4362354/

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