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

19 Dec 2012

Abstract excerpt

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 generated iPSCs from SMA patients (SMA-iPSCs) using nonviral, nonintegrating episomal vectors and used a targeted gene...

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PMID: 23253609
DOI: 10.1126/scitranslmed.3004108

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