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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Detalles Bibliográficos
Autores principales: Lange, Amy M, Altynova, Ekaterina S, Nguyen, Giang N, Sabatino, Denise E
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5040173/
https://www.ncbi.nlm.nih.gov/pubmed/27738645
http://dx.doi.org/10.1038/mtm.2016.64

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5040173/
https://www.ncbi.nlm.nih.gov/pubmed/27738645
http://dx.doi.org/10.1038/mtm.2016.64

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