Generation of spinocerebellar ataxia type 3 patient-derived induced pluripotent stem cell line (CSUXHi005-A) from human urine epithelial cells

Lang He; Xiaobo Han; Huifang Zhao; Shuai Li; Zhao Chen; Chunrong Wang; Shang Wang; Linliu Peng; Weiyue Deng; Zhiyuan Li; Beisha Tang; Hong Jiang

doi:10.1016/j.scr.2021.102289

Generation of spinocerebellar ataxia type 3 patient-derived induced pluripotent stem cell line (CSUXHi005-A) from human urine epithelial cells

Stem Cell Res. 2021 May:53:102289. doi: 10.1016/j.scr.2021.102289. Epub 2021 Mar 17.

Authors

Lang He¹, Xiaobo Han², Huifang Zhao³, Shuai Li², Zhao Chen⁴, Chunrong Wang⁵, Shang Wang¹, Linliu Peng¹, Weiyue Deng⁶, Zhiyuan Li⁷, Beisha Tang⁸, Hong Jiang⁹

Affiliations

¹ Department of Neurology, Xiangya Hospital, Central South University, Changsha, Hunan, China.
² University of Chinese Academy of Sciences, Beijing, China.
³ School of Life Sciences, University of Science and Technology of China, Hefei, China.
⁴ Department of Neurology, Xiangya Hospital, Central South University, Changsha, Hunan, China; Key Laboratory of Hunan Province in Neurodegenerative Disorders, Central South University, Changsha, Hunan, China; National Clinical Research Center for Geriatric Diseases, Central South University, Changsha, Hunan, China.
⁵ Department of Pathology, Xiangya Hospital, Central South University, Changsha, China.
⁶ Department of Anatomy and Neurobiology, Xiangya School of Medicine, Central South. University, Changsha, Hunan, China.
⁷ University of Chinese Academy of Sciences, Beijing, China; School of Life Sciences, University of Science and Technology of China, Hefei, China; Department of Anatomy and Neurobiology, Xiangya School of Medicine, Central South. University, Changsha, Hunan, China; CAS Key Laboratory of Regenerative Biology, Guangdong Provincial Key Laboratory of Stem Cell and Regenerative Medicine, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou, China; GZMU-GIBH Joint School of Life Sciences, Guangzhou Medical University, Guangzhou, China.
⁸ Department of Neurology, Xiangya Hospital, Central South University, Changsha, Hunan, China; Key Laboratory of Hunan Province in Neurodegenerative Disorders, Central South University, Changsha, Hunan, China; National Clinical Research Center for Geriatric Diseases, Central South University, Changsha, Hunan, China; Laboratory of Medical Genetics, Central South University, Changsha, Hunan, China.
⁹ Department of Neurology, Xiangya Hospital, Central South University, Changsha, Hunan, China; Key Laboratory of Hunan Province in Neurodegenerative Disorders, Central South University, Changsha, Hunan, China; National Clinical Research Center for Geriatric Diseases, Central South University, Changsha, Hunan, China; Laboratory of Medical Genetics, Central South University, Changsha, Hunan, China; School of Basic Medical Science, Central South University, Changsha, Hunan, China. Electronic address: [email protected].

PMID: 33761381
DOI: 10.1016/j.scr.2021.102289

Abstract

Urine epithelial cells were harvested from a 32-year old female patient with spinocerebellar ataxia type 3 (SCA3) and reprogrammed into induced pluripotent stem cells (iPSCs) by non-integration system. The SCA3 derived iPSCs line, CSUXHi005-A, maintained 76 CAG expansions in the ATXN3 gene, was characterized by the expression of pluripotency markers and normal karyotype. The newly generated iPSCs retain the ability to differentiate into three germ layers by teratoma test, which provide an ideal tool for disease modeling, drug screening, and cellular therapy.

Publication types

Case Reports
Research Support, Non-U.S. Gov't

MeSH terms

Adult
Ataxin-3
Cell Differentiation
Epithelial Cells
Female
Humans
Induced Pluripotent Stem Cells*
Machado-Joseph Disease*

Substances

Ataxin-3