Correcting CFTR: New Gene Editing Strategies for Rescuing CFTR Function Ex Vivo

被引：1
|
作者：
Ryan, Amy L. [1 ,2 ]
机构：
[1] Univ Southern Calif, Dept Med, Div Pulm Crit Care & Sleep Med, Hastings Ctr Pulm Res, Los Angeles, CA 90007 USA
[2] Univ Southern Calif, Dept Stem Cell Biol & Regenerat Med, Los Angeles, CA 90007 USA
来源：
CELL STEM CELL | 2020年 / 26卷 / 04期
关键词：
CYSTIC-FIBROSIS; CELLS;
D O I：
10.1016/j.stem.2020.03.012
中图分类号：
Q813 [细胞工程];
学科分类号：
摘要：
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.
引用
收藏
页码：476 / 478
页数：3
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