Phenotypic correction of murine hemophilia A using an iPS cell-based therapy

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...

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Autori principali: Xu, Dan, Alipio, Zaida, Fink, Louis M., Adcock, Dorothy M., Yang, Jianchang, Ward, David C., Ma, Yupo
Natura: Artigo
Lingua:Inglês
Pubblicazione: National Academy of Sciences 2009
Soggetti:
Biological Sciences
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC2630078/
https://ncbi.nlm.nih.gov/pubmed/19139414
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0812090106
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