Unknown,Transcriptomics,Genomics,Proteomics

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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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Publications


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]

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