Nanocapsule-delivered Sleeping Beauty mediates therapeutic Factor VIII expression in liver sinusoidal endothelial cells of hemophilia A mice

Liver sinusoidal endothelial cells are a major endogenous source of Factor VIII (FVIII), lack of which causes the human congenital bleeding disorder hemophilia A. Despite extensive efforts, gene therapy using viral vectors has shown little success in clinical hemophilia trials. Here we achieved cell...

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Autors principals: Kren, Betsy T., Unger, Gretchen M., Sjeklocha, Lucas, Trossen, Alycia A., Korman, Vicci, Diethelm-Okita, Brenda M., Reding, Mark T., Steer, Clifford J.
Format: Artigo
Idioma:Inglês
Publicat: American Society for Clinical Investigation 2009
Matèries:
Technical Advance
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC2701853/
https://ncbi.nlm.nih.gov/pubmed/19509468
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI34332
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