利用诱导型慢病毒载体从一名患有CRB1视网膜色素变性患者的外周血细胞中生成诱导多能干细胞系ZOCi001-A。
Using inducible lentiviral vectors to generate induced pluripotent stem cell line ZOCi001-A from peripheral blood cells of a patient with CRB1 retinitis pigmentosa.
作者信息
Tang Xiangcheng, Liu Xinyu, Chen Zhigang, Luo Lixia, Liu Xialin, Deng Juan, Li David Wan-Cheng, Liu Yizhi
机构信息
State Key Laboratory of Ophthalmology, Zhongshan Ophthalmic Center, Sun Yat-Sen University, Guangzhou, Guangdong, 510060, China.
Third Affiliated Hospital of Sun Yat-sen University, Guangzhou, Guangdong, 510630, China.
出版信息
Stem Cell Res. 2020 May;45:101817. doi: 10.1016/j.scr.2020.101817. Epub 2020 Apr 30.
We isolated peripheral blood mononuclear cells from a patient diagnosed with early-onset non-syndromic retinitis pigmentosa caused by compound heterozygous mutations in CRB1 (NM_001257965): c.1369C>T (p.R457X) and c.2027C>T (p.T676M). These cells were transfected with four inducible plasmids encoding human OCT4, SOX2, KLF4 or C-MYC together. Transfected cells were induced to form pluripotent stem cells (iPSCs) expressing the pluripotent stem cell markers SOX2, OCT4, KLF4, SSEA4 and NANOG and presenting a normal karyotype. These cells could be differentiated into three germ layers as well as retinal organoids, and thus provide a valuable cellular model for the study of development of major retina diseases.
我们从一名被诊断患有由CRB1（NM_001257965）复合杂合突变引起的早发性非综合征性视网膜色素变性的患者中分离出外周血单个核细胞：c.1369C>T（p.R457X）和c.2027C>T（p.T676M）。将这些细胞与四种分别编码人OCT4、SOX2、KLF4或C-MYC的可诱导质粒一起转染。转染后的细胞被诱导形成表达多能干细胞标志物SOX2、OCT4、KLF4、SSEA4和NANOG且核型正常的多能干细胞（iPSC）。这些细胞可分化为三个胚层以及视网膜类器官，因此为研究主要视网膜疾病的发展提供了有价值的细胞模型。
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