A MicroRNA-regulated and GP64-pseudotyped Lentiviral Vector Mediates Stable Expression of FVIII in a Murine Model of Hemophilia A

The objective to use gene therapy to provide sustained, therapeutic levels of factor VIII (FVIII) for hemophilia A is compromised by the emergence of inhibitory antibodies that prevent FVIII from performing its essential function as a cofactor for factor IX (FIX). FVIII appears to be more immunogeni...

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Detalhes bibliográficos
Main Authors: Matsui, Hideto, Hegadorn, Carol, Ozelo, Margareth, Burnett, Erin, Tuttle, Angie, Labelle, Andrea, McCray, Paul B, Naldini, Luigi, Brown, Brian, Hough, Christine, Lillicrap, David
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2011
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3070093/
https://ncbi.nlm.nih.gov/pubmed/21285959
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.290
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