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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Detalhes bibliográficos
Publicado no: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.
Formato: Artigo
Idioma:Inglês
Publicado em: 2012
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Article
Acesso em linha: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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