Format

Send to

Choose Destination
Stem Cell Res. 2019 Dec;41:101564. doi: 10.1016/j.scr.2019.101564. Epub 2019 Aug 29.

Generation of induced pluripotent stem cell line (ZZUi0014-A) from a patient with spinocerebellar ataxia type 3.

Author information

1
Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou 450052, China.
2
Zhengzhou University, Zhengzhou, Henan 450001, China.
3
School of life sciences, Zhengzhou University, Zhengzhou, Henan 450001, China.
4
Center for Stem Cell and Regenerative Medicine, Department of Basic Medical Sciences, The First Affiliated Hospital, College of Medicine, Zhejiang University, Hangzhou, Zhejiang 310058, China; Institute of Hematology, Zhejiang University, Hangzhou, Zhejiang 310058, China.
5
Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou 450052, China. Electronic address: xuyuming@zzu.edu.cn.
6
Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou 450052, China. Electronic address: shichanghe@gmail.com.

Abstract

Spinocerebellar ataxia type 3 (SCA3) is an autosomal dominant disorder that is caused by the abnormal amplification of cytosine-adenine-guanine (CAG) trinucleotide repeats in the ATXN3 gene. The main feature of SCA3 is progressive ataxia. Currently, no effective treatment exists for this condition. For this study, we obtained dermal fibroblasts from a patient. The fibroblasts were successfully transformed into induced pluripotent stem cells (iPSCs) by employing episomal plasmids expressing OCT3/4, SOX2, KLF4, LIN28, and L-MYC. Our approach offers a resource for further research into SCA3 mechanism in an attempt to facilitate the development and screening of pharmaceutical and gene therapy.

PMID:
31639609
DOI:
10.1016/j.scr.2019.101564
Free full text

Supplemental Content

Full text links

Icon for Elsevier Science
Loading ...
Support Center