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CRISPR/Cas9-mediated targeted gene correction in amyotrophic lateral sclerosis patient iPSCs

11 Apr 2017

Abstract excerpt

Amyotrophic lateral sclerosis (ALS) is a complex neurodegenerative disease with cellular and molecular mechanisms yet to be fully described. Mutations in a number of genes including SOD1 and FUS are associated with familial ALS. Here we report the generation of induced pluripotent stem cells (iPSCs) from fibroblasts of familial ALS patients bearing SOD1+/A272C and FUS+/G1566A mutations, respectively. We further...

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PMID: 28401346
DOI: 10.1007/s13238-017-0397-3

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