利用CRISPR/Cas9从多囊肾病诱导多能干细胞系中生成基因校正的人类同基因IBMS-iPSC-014-C。
Generation of a gene corrected human isogenic IBMS-iPSC-014-C from polycystic-kidney-disease induced pluripotent stem cell line using CRISPR/Cas9.
作者信息
Liu Chun-Lin, Huang Ching-Ying, Chen Hung-Chih, Lu Huai-En, Hsieh Patrick C H, Lee Jia-Jung
机构信息
Human Disease iPSC Service Consortium, Institute of Biomedical Sciences, Academia Sinica, Taipei, Taiwan; Institute of Biomedical Sciences, Academia Sinica, Taipei, Taiwan.
Institute of Biomedical Sciences, Academia Sinica, Taipei, Taiwan.
出版信息
Stem Cell Res. 2020 May;45:101784. doi: 10.1016/j.scr.2020.101784. Epub 2020 Apr 20.
We report the engendering an isogenic iPSC line from the IBMS-iPSC-014-05 with homozygous correction of the R803X, Chr4: 88989098C > T in PKD2, using CRISPR/Cas9 technology. The results from the isogenic control, IBMS-iPSC-014-05C, showed that mutation had been corrected, while maintaining normal morphology, pluripotency, and differentiation capacity into three germ layers.
我们报告了利用CRISPR/Cas9技术从IBMS-iPSC-014-05产生了一个等基因诱导多能干细胞系，该系对PKD2基因中第4号染色体88989098位的C>T（R803X）进行了纯合校正。等基因对照IBMS-iPSC-014-05C的结果表明，突变已得到校正，同时保持了正常形态、多能性以及向三个胚层分化的能力。
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