Genetic Correction of Human Induced Pluripotent Stem Cells from Patients with Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is among the most common genetic neurological diseases that cause infant mortality. Induced pluripotent stem cells (iPSCs) generated from skin fibroblasts from SMA patients and genetically corrected have been proposed to be useful for autologous cell therapy. We generat...

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發表在:Sci Transl Med
Main Authors: Corti, Stefania, Nizzardo, Monica, Simone, Chiara, Falcone, Marianna, Nardini, Martina, Ronchi, Dario, Donadoni, Chiara, Salani, Sabrina, Riboldi, Giulietta, Magri, Francesca, Menozzi, Giorgia, Bonaglia, Clara, Rizzo, Federica, Bresolin, Nereo, Comi, Giacomo P.
格式: Artigo
語言:Inglês
出版: 2012
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Article
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC4722730/
https://ncbi.nlm.nih.gov/pubmed/23253609
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.3004108
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