Functional rescue of F508del-CFTR through revertant mutations introduced by CRISPR base editing
Irene Carrozzo, Giulia Maule, Carmelo Gentile, Alessandro Umbach, Matteo Ciciani, Daniela Guidone, Martina De Santis, Gianluca Petris, Luis J. V. Galietta, Daniele Arosio, Anna Cereseto
This study uses CRISPR base editing to introduce revertant mutations in F508del-CFTR, rescuing protein folding and function in CF patient-derived cells.
Molecular Therapy · Jan 9, 2025Peer-reviewedBioengineering