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Correction of murine β-thalassemia after minimal lentiviral gene transfer and homeostatic in vivo erythroid expansion

A challenge for gene therapy of genetic diseases is to maintain corrected cell populations in subjects undergoing transplantation in cases in which the corrected cells do not have intrinsic selective advantage over nontransduced cells. For inherited hematopoietic disorders, limitations include ineff...

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Dettagli Bibliografici
Autori principali: Negre, Olivier, Fusil, Floriane, Colomb, Charlotte, Roth, Shoshannah, Gillet-Legrand, Beatrix, Henri, Annie, Beuzard, Yves, Bushman, Frederic, Leboulch, Philippe, Payen, Emmanuel
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Hematology 2011
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3109707/
https://ncbi.nlm.nih.gov/pubmed/21436071
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2010-01-263582
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