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CRISPR/Cas9 mediated gene correction ameliorates abnormal phenotypes in spinocerebellar ataxia type 3 patient-derived induced pluripotent stem cells.

Translational psychiatry - 17 Sept 2021

He Lang, Wang Shang, Peng Linliu, Zhao Huifang, Li Shuai, Han Xiaobo, Habimana Jean de Dieu, Chen Zhao, Wang Chunrong, Peng Yun, Peng Huirong, Xie Yue, Lei Lijing, Deng Qi, Wan Linlin, Wan Na, Yuan Hongyu, Gong Yiqing, Zou Guangdong, Li Zhiyuan, Tang Beisha, Jiang Hong

Abstract excerpt

Spinocerebellar ataxia type 3/Machado-Joseph disease (SCA3/MJD) is a progressive autosomal dominant neurodegenerative disease caused by abnormal CAG repeats in the exon 10 of ATXN3. The accumulation of the mutant ataxin-3 proteins carrying expanded polyglutamine (polyQ) leads to selective degeneration of neurons. Since the pathogenesis of SCA3 has not been fully elucidated, and no effective therapies have been...

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PMID: 34535635
DOI: 10.1038/s41398-021-01605-2
PMCID: PMC8448778

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