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

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 degenerati...

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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8448778/
https://www.ncbi.nlm.nih.gov/pubmed/34535635
http://dx.doi.org/10.1038/s41398-021-01605-2

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8448778/
https://www.ncbi.nlm.nih.gov/pubmed/34535635
http://dx.doi.org/10.1038/s41398-021-01605-2

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