CRISPR/Cas9 Mediated Gene Correction Ameliorates Abnormal Phenotypes in Spinocerebellar Ataxia Type 3 Patient-Derived Induced Pluripotent Stem Cells

Spinocerebellar ataxia type 3 (SCA3)-iPSCs could be corrected by the replacement of the abnormal CAG expansions with normal repeats using CRISPR/Cas9-mediated homologous recombination strategy. Corrected SCA3-iPSCs retained pluripotent and normal karyotype, which could be differentiated into a neural stem cell and neuronal cells.