Generation of induced pluripotent stem cell line (ZZUi004-A) from urine sample of a patient with spinocerebellar ataxia type 3
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. Copyright © 2018. Published by Elsevier B.V.
|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|
|Journal||Stem cell research|
|Publication Date||2018 Apr;28:71-74|