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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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Detalles Bibliográficos
Autores principales:	Brown, Harrison C, Wright, J Fraser, Zhou, Shangzhen, Lytle, Allison M, Shields, Jordan E, Spencer, H Trent, Doering, Christopher B
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2014
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4362354/ https://www.ncbi.nlm.nih.gov/pubmed/26015976 http://dx.doi.org/10.1038/mtm.2014.36

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