Functional rescue of F508del-CFTR through revertant mutations introduced by CRISPR base editing.
通过 CRISPR 碱基编辑引入回复突变，实现 F508del-CFTR 的功能恢复
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2025.01.011
Carrozzo Irene, Maule Giulia, Gentile Carmelo, Umbach Alessandro, Ciciani Matteo, Guidone Daniela, De Santis Martina, Petris Gianluca, Vicente Galietta Luis Juan, Arosio Daniele, Cereseto Anna
