GeneticsJCI InsightPeer-reviewed
Base editing and nanoparticle transfection of airway cell types essential for treatment of cystic fibrosis
Erin W. Kavanagh, Anya T. Joynt, A. Pion…·Garry R. Cutting
This study corrects a CFTR splice site variant in airway cells using base editors and nanoparticles, showing increased CFTR expression and functional rescue in multiple cell types.