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

被引：13
|
作者：
Wang, Yanlin [1 ]
Sun, Huifang [1 ]
Wang, Zhuoya [1 ]
Yue, Yiwei [2 ]
Zhang, Rui [1 ]
Yang, Jing [1 ]
Liu, Yutao [1 ]
Liu, Han [1 ]
Zhang, Qi [1 ]
Zhang, Shoutao [3 ]
Zhang, Jin [4 ,5 ]
Xu, Yuming [1 ]
Shi, Changhe [1 ]
机构：
[1] Zhengzhou Univ, Affiliated Hosp 1, Dept Neurol, Zhengzhou 450052, Peoples R China
[2] Zhengzhou Univ, Zhengzhou 450001, Henan, Peoples R China
[3] Zhengzhou Univ, Sch Life Sci, Zhengzhou 450001, Henan, Peoples R China
[4] Zhejiang Univ, Affiliated Hosp 1, Dept Basic Med Sci, Ctr Stem Cell & Regenerat Med,Coll Med, Hangzhou 310058, Zhejiang, Peoples R China
[5] Zhejiang Univ, Inst Hematol, Hangzhou 310058, Zhejiang, Peoples R China
来源：
STEM CELL RESEARCH | 2019年 / 41卷
基金：
国家重点研发计划; 中国国家自然科学基金;
关键词：
D O I：
10.1016/j.scr.2019.101564
中图分类号：
Q813 [细胞工程];
学科分类号：
摘要：
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.
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页数：4
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