从一名3型脊髓小脑共济失调患者的尿液样本中生成诱导多能干细胞系（ZZUi004-A）。
Generation of induced pluripotent stem cell line (ZZUi004-A) from urine sample of a patient with spinocerebellar ataxia type 3.
作者信息
Wang Yanlin, Shi Changhe, Wang Zhilei, Sun Huifang, Yang Zhihua, Zhang Fan, Liu Yutao, Liu Han, Jiang Chenyang, Zhang Shoutao, Xu Yuming, Wen Xuejun
机构信息
Department of Neurology, The First affiliated Hospital of Zhengzhou University, Zhengzhou University, Zhengzhou, Henan 450052, China.
Department of Geriatrics, The Second affiliated Hospital of Zhengzhou University, Zhengzhou 450014, Henan, China.
出版信息
Stem Cell Res. 2018 Apr;28:71-74. doi: 10.1016/j.scr.2018.01.027. Epub 2018 Jan 31.
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.
3型脊髓小脑共济失调（SCA3）是一种由ATXN3基因区域CAG重复序列扩增引起的常染色体显性遗传神经退行性疾病。SCA3的主要特征是进行性共济失调，影响平衡、步态和言语。通过使用仙台病毒递送系统，一名SCA3患者的尿液细胞（UCs）被成功重编程为诱导多能干细胞（iPSCs）。ZZUi004-A细胞系可能为进一步研究SCA3发病机制以及药物测试和基因治疗研究提供一个有力的平台。
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