BioengineeringMolecular TherapyPeer-reviewed
Functional rescue of F508del-CFTR through revertant mutations introduced by CRISPR base editing
Irene Carrozzo, Giulia Maule, Carmelo Gentile…·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.