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Gene Expression Analysis of native and disease-corrected motor neurons from human spinal muscular atrophy induced pluripotent stem cells free of vector and transgenic sequences

ABSTRACT: This SuperSeries is composed of the SubSeries listed below. Refer to individual Series

ORGANISM(S): Homo sapiens

SUBMITTER: Stefania Corti

PROVIDER: E-GEOD-27207 | biostudies-arrayexpress |

REPOSITORIES: biostudies-arrayexpress

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Genetic correction of human induced pluripotent stem cells from patients with spinal muscular atrophy.

Corti Stefania S Nizzardo Monica M Simone Chiara C Falcone Marianna M Nardini Martina M Ronchi Dario D Donadoni Chiara C Salani Sabrina S Riboldi Giulietta G Magri Francesca F Menozzi Giorgia G Bonaglia Clara C Rizzo Federica F Bresolin Nereo N Comi Giacomo P GP

Science translational medicine 20121201 165

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 correction approach based on single-stranded oligonucleotides to convert the survival mo ...[more]

PMID: 23253609

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