Allele-Specific Targeting of Mutant Ataxin-3 by Antisense Oligonucleotides in SCA3-iPSC-Derived Neurons

The authors provided proof of principle that allele-specific lowering of polyQ-expanded ataxin-3 by selective antisense oligonucleotides was feasible and long lasting with sparing of wildtype ataxin-3 expression in a human cell culture model that was genetically identical to spinocerebellar ataxia type 3 (SCA3) patients.
[Molecular Therapy-Nucleic Acids]
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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.
[Translational Psychiatry]
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