当前位置: X-MOL 学术Cell Stem Cell › 论文详情
Our official English website, www.x-mol.net, welcomes your feedback! (Note: you will need to create a separate account there.)
Correcting CFTR: New Gene Editing Strategies for Rescuing CFTR Function Ex Vivo
Cell Stem Cell ( IF 19.8 ) Pub Date : 2020-04-02 , DOI: 10.1016/j.stem.2020.03.012
Amy L. Ryan

Gene correction presents one of few options for a cure for all patients with cystic fibrosis. This commentary discusses new applications of CRISPR-based gene editing technology with increased efficiency and specificity to correct the cystic fibrosis transmembrane regulator (CFTR) function in patient-specific primary epithelial cells.



中文翻译:

纠正CFTR:新基因编辑策略抢救CFTR功能离体

基因校正是治愈所有囊性纤维化患者的少数选择之一。本文评论了基于CRISPR的基因编辑技术的新应用,该技术具有更高的效率和特异性,可纠正患者特异性原发性上皮细胞中的囊性纤维化跨膜调节剂(CFTR)功能。

更新日期:2020-04-20
down
wechat
bug