Base editing and nanoparticle transfection of airway cell types essential for treatment of cystic fibrosis
Erin W. Kavanagh, Anya T. Joynt, A. Pion, Alice C. Eastman, Alianna I. Parr, K. Starego, Manav Jain, Sydney R. Shannon, Edwin Yoo, Gregory A. Newby, Stephany Yi Tzeng, Neeraj Sharma, Jordan J. Green, 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.
JCI Insight · May 7, 2026Peer-reviewedGenetics