从人诱导多能干细胞系生成CFTR基因敲入报告细胞系（MHHi006-A-1）。
Generation of a CFTR knock-in reporter cell line (MHHi006-A-1) from a human induced pluripotent stem cell line.
作者信息
Engels Lena, Olmer Ruth, de la Roche Jeanne, Göhring Gudrun, Ulrich Saskia, Haller Ralf, Martin Ulrich, Merkert Sylvia
机构信息
Leibniz Research Laboratories for Biotechnology and Artificial Organs (LEBAO), Department of Cardiothoracic, Transplantation and Vascular Surgery, Hannover Medical School, 30625 Hannover, Germany; REBIRTH-Cluster of Excellence, Hannover Medical School, 30625 Hannover, Germany; Biomedical Research in Endstage and Obstructive Lung Disease (BREATH), German Center for Lung Research (DZL), Germany.
Institute for Neurophysiology, Hannover Medical School, 30625 Hannover, Germany.
出版信息
Stem Cell Res. 2019 Oct;40:101542. doi: 10.1016/j.scr.2019.101542. Epub 2019 Aug 20.
CFTR encodes for a chloride ion channel expressed primarily in secretory epithelia in the airways, intestine, liver and other tissues. Mutations in the CFTR gene have been identified in people suffering from Cystic Fibrosis. Here, we established a CFTR knock-in reporter cell line from a human iPSC line (MHHi006-A) using TALEN technology. The reporter enables the monitoring and optimization of the differentiation of pluripotent stem cells into CFTR expressing epithelia on a single cell level, as well as the enrichment of CFTR positive cells, which represent an excellent tool for Cystic Fibrosis disease modelling, drug screening and ultimately cellular therapies.
CFTR编码一种主要在气道、肠道、肝脏和其他组织的分泌上皮中表达的氯离子通道。在患有囊性纤维化的人群中已鉴定出CFTR基因突变。在此，我们使用TALEN技术从人诱导多能干细胞系（MHHi006-A）建立了一个CFTR敲入报告细胞系。该报告基因能够在单细胞水平上监测和优化多能干细胞向表达CFTR的上皮细胞的分化，以及富集CFTR阳性细胞，这为囊性纤维化疾病建模、药物筛选以及最终的细胞治疗提供了一个出色的工具。
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