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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Detaylı Bibliyografya
Asıl Yazarlar: Kren, Betsy T., Unger, Gretchen M., Sjeklocha, Lucas, Trossen, Alycia A., Korman, Vicci, Diethelm-Okita, Brenda M., Reding, Mark T., Steer, Clifford J.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: American Society for Clinical Investigation 2009
Konular:
Technical Advance
Online Erişim: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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