Shanghai Mental Health Center, Shanghai Jiao Tong University School of Medicine, Shanghai 200030, PR China.
Bioland Laboratory (Guangzhou Regenerative Medicine and Health Guangdong Laboratory), Guangzhou 510005, PR China; CAS Key Laboratory of Regenerative Biology, Guangdong Provincial Key Laboratory of Stem Cell and Regenerative Medicine, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou 510530, PR China.
Stem Cell Res. 2021 Apr;52:102227. doi: 10.1016/j.scr.2021.102227. Epub 2021 Feb 11.
Tourette's syndrome (TS) is a childhood onset neuropsychiatric disorder. The underlying pathogenesis of TS remains unclear. A human induced pluripotent stem cell (hiPSC) line, derived from the peripheral blood mononuclear cells (PBMCs) from a TS patient, was generated by the reprogramming with episomal vectors expressing OCT4, SOX2, KLF4 and c-MYC. The generated hiPSC line is free of episomal vectors, with normal karyotype, expressing pluripotency markers and can differentiate into three germ layers in vivo. This hiPSC line from TS patient can offer a useful resource for studying the pathological mechanism of TS and screening effective drugs for therapeutic purpose.
妥瑞氏症（TS）是一种儿童期发病的神经精神疾病。TS 的潜在发病机制尚不清楚。我们通过转染表达 OCT4、SOX2、KLF4 和 c-MYC 的附加型载体，从 TS 患者的外周血单核细胞（PBMCs）中生成了一个人诱导多能干细胞（hiPSC）系。生成的 hiPSC 系不含附加型载体，具有正常核型，表达多能性标记物，并能在体内分化为三个胚层。该 TS 患者来源的 hiPSC 系为研究 TS 的病理机制和筛选有效的治疗药物提供了有用的资源。
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