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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Detalhes bibliográficos

Principais autores:	Harrison C Brown, J Fraser Wright, Shangzhen Zhou, Allison M Lytle, Jordan E Shields, H Trent Spencer, Christopher B Doering
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Elsevier 2014-01-01
coleção:	Molecular Therapy: Methods & Clinical Development
Acesso em linha:	http://www.sciencedirect.com/science/article/pii/S2329050116301036
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