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：

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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。