Article
Phenotypic correction of murine hemophilia A using an iPS cell-based therapy.
Proceedings of the National Academy of Sciences of the United States of America - 20 Jan 2009
Xu Dan, Alipio Zaida, Fink Louis M, Adcock Dorothy M, Yang Jianchang, Ward David C, Ma Yupo
Abstract excerpt
Hemophilia A is caused by mutations within the Factor VIII (FVIII) gene that lead to depleted protein production and inefficient blood clotting. Several attempts at gene therapy have failed for various reasons-including immune rejection. The recent generation of induced pluripotent stem (iPS) cells from somatic cells by the ectopic expression of 3 transcription factors, Oct4, Sox2, and Klf4, provides a means of...
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