Generation of spinocerebellar ataxia type 3 patient-derived induced pluripotent stem cell line (CSUXHi005-A) from human urine epithelial cells
Generation of spinocerebellar ataxia type 3 patient-derived induced pluripotent stem cell line (CSUXHi005-A) from human urine epithelial cells
复制标题
从人尿上皮细胞生成脊髓小脑共济失调 3 型患者来源的诱导多能干细胞系 (CSUXHi005-A)
DOI：
10.1016/j.scr.2021.102289
复制
发表时间：
2021-03-21
影响因子：
1.2
通讯作者：
Jiang, Hong
中科院分区：
文献类型：
--
作者：
He, Lang;Han, Xiaobo;Jiang, Hong
取1例32岁女性脊髓小脑型共济失调(SCA3)患者的尿液上皮细胞，用非整合系统重编程为诱导多能干细胞(IPSCs)。SCA3来源的IPSCs系CSUXHi005-A在ATXN3基因上保持76个CAG扩增，具有多潜能标记的表达和正常的核型。新生成的IPSCs通过畸胎瘤试验保持了分化为三个胚层的能力，这为疾病建模、药物筛选和细胞治疗提供了理想的工具。
Urine epithelial cells were harvested from a 32-year old female patient with spinocerebellar ataxia type 3 (SCA3) and reprogrammed into induced pluripotent stem cells (iPSCs) by non-integration system. The SCA3 derived iPSCs line, CSUXHi005-A, maintained 76 CAG expansions in the ATXN3 gene, was characterized by the expression of pluripotency markers and normal karyotype. The newly generated iPSCs retain the ability to differentiate into three germ layers by teratoma test, which provide an ideal tool for disease modeling, drug screening, and cellular therapy.