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Generation of induced pluripotent stem cell line (ZZUi004-A) from urine sample of a patient with spinocerebellar ataxia type 3.

Abstract：

:Spinocerebellar ataxia type 3 (SCA3) is a dominantly inherited neurodegenerative disease caused by a CAG repeat expansion in the region of the ATXN3 gene. The main feature of SCA3 is progressive ataxia, which affects balance, gait, and speech. Urine cells (UCs) of a SCA3 patient were successfully translated to induced pluripotent stem cells (iPSCs) by using the Sendai virus delivery system. ZZUi004-A cell line may provide a robust platform for further study of SCA3 pathogenesis as well as drug testing and gene therapy research.

journal_name

Stem Cell Res

journal_title

Stem cell research

authors

Wang Y,Shi C,Wang Z,Sun H,Yang Z,Zhang F,Liu Y,Liu H,Jiang C,Zhang S,Xu Y,Wen X

doi

10.1016/j.scr.2018.01.027

subject

Has Abstract

pub_date

2018-04-01 00:00:00

pages

71-74

eissn

1873-5061

issn

1876-7753

pii

S1873-5061(18)30030-8

journal_volume

28

pub_type

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