Using inducible lentiviral vectors to generate induced pluripotent stem cell line ZOCi001-A from peripheral blood cells of a patient with CRB1 -/- retinitis pigmentosa
Using inducible lentiviral vectors to generate induced pluripotent stem cell line ZOCi001-A from peripheral blood cells of a patient with CRB1 -/- retinitis pigmentosa
复制标题
使用诱导型慢病毒载体从 CRB1 -/- 视网膜色素变性患者的外周血细胞中产生诱导多能干细胞系 ZOCi001-A
DOI：
10.1016/j.scr.2020.101817
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发表时间：
2020
影响因子：
1.2
通讯作者：
Yizhi Liu
中科院分区：
文献类型：
--
作者：
Xiangcheng Tang;Xinyu Liu;Zhigang Chen;Lixia Luo;Xialin Liu;Juan Deng;David Wan-Cheng Li;Yizhi Liu
我们从诊断为早发性非综合征性视网膜色素变性的患者中分离外周血单核细胞，该患者由CRB 1（NM_001257965）中的复合杂合突变引起：c.1369C>T（p.R457X）和c.2027C>T（p.T676M）。这些细胞用一起编码人OCT 4、SOX 2、KLF 4或C-MYC的四种诱导型质粒转染。转染的细胞被诱导形成表达多能干细胞标志物S 0X 2、0 CT 4、KLF 4、SSEA 4和NANOG并呈现正常核型的多能干细胞（iPSC）。这些细胞可以分化为三个胚层以及视网膜类器官，从而为研究主要视网膜疾病的发展提供了有价值的细胞模型。
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.