Ding Yinjuan, Wang Long, Ji Weiping, Chen Zhanguo, Wang Dexuan, Chen Congde, Tong Hongfei, Han Zhao, Niu Chao, Chu Maoping, Huang Jinyu, Guo Xiaoling
Center of Scientific Research, the Second Affiliated Hospital and Yuying Children's Hospital of Wenzhou Medical University, Wenzhou, Zhejiang, China.
Center of Scientific Research, the Second Affiliated Hospital and Yuying Children's Hospital of Wenzhou Medical University, Wenzhou, Zhejiang, China; Department of Cardiology, the Affiliated Hangzhou First People's Hospital of Zhejiang University School of Medicine, Hangzhou, Zhejiang, China.
Stem Cell Res. 2020 Dec;49:102064. doi: 10.1016/j.scr.2020.102064. Epub 2020 Oct 27.
Human induced pluripotent stem (iPS) cells expressing Cas9 protein are valuable for the pathogenic mechanism study and drug discovery. These cells can be efficiently induced to differentiate into disease cell models with specific mutations through adding designed sgRNAs. Here, we generated a human gene-editable iPS cell line by gene editing method that Cas9 gene driven by Tet-on operator was perfectly integrated into the human AAVS1 safe harbor locus. The established Cas9 expression iPS cell line named as WMUi013-A can express endogenous pluripotent markers, has the ability to differentiate into the three germ layers, and possesses a normal karyotype.
表达Cas9蛋白的人诱导多能干细胞（iPS细胞）对于致病机制研究和药物发现具有重要价值。通过添加设计好的sgRNA，这些细胞可以被高效诱导分化为具有特定突变的疾病细胞模型。在此，我们通过基因编辑方法构建了一种人类基因可编辑的iPS细胞系，即由Tet-on启动子驱动的Cas9基因完美整合到人类AAVS1安全位点。所建立的表达Cas9的iPS细胞系命名为WMUi013-A，它能表达内源性多能性标志物，具有分化为三个胚层的能力，并且拥有正常的核型。
