Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells

Realizing the therapeutic potential of human induced pluripotent stem (iPS) cells will require robust, precise and safe strategies for genetic modification, as cell therapies that rely on randomly integrated transgenes pose oncogenic risks. Here we describe a strategy to genetically modify human iPS...

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Detalhes bibliográficos
Main Authors: Papapetrou, Eirini P, Lee, Gabsang, Malani, Nirav, Setty, Manu, Riviere, Isabelle, Tirunagari, Laxmi M S, Kadota, Kyuichi, Roth, Shoshannah L, Giardina, Patricia, Viale, Agnes, Leslie, Christina, Bushman, Frederic D, Studer, Lorenz, Sadelain, Michel
Formato: Artigo
Idioma:Inglês
Publicado em: 2010
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3356916/
https://ncbi.nlm.nih.gov/pubmed/21151124
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/nbt.1717
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