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Overexpression of factor VIII after AAV delivery is transiently associated with cellular stress in hemophilia A mice

Factor VIII (FVIII) is a large glycoprotein that is challenging to express both in vitro and in vivo. Several studies suggest that high levels of FVIII expression can lead to cellular stress. After gene transfer, transgene expression is restricted to a subset of cells and the increased FVIII load pe...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Lange, Amy M, Altynova, Ekaterina S, Nguyen, Giang N, Sabatino, Denise E
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5040173/
https://ncbi.nlm.nih.gov/pubmed/27738645
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2016.64
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