Systemic in utero gene editing as a treatment for cystic fibrosis
Adele S. Ricciardi, Christina Barone, Rachael Putman, Elias Quijano, Anisha Gupta, Richard Nguyen, Hanna K. Mandl, Alexandra S. Piotrowski-Daspit, Francesc López‐Giráldez, Valerie L. Luks, Mollie R. Freedman-Weiss, James S. Farrelly, Samantha L. Ahle, Anna Y. Lynn, Peter M. Glazer, , Marie E. Egan
This study demonstrates systemic in utero gene editing using PNAs in nanoparticles to correct CFTR mutations in multiple organs of CF mice, showing sustained CFTR activity without off-target effects.
Proceedings of the National Academy of Sciences · Jun 10, 2025Peer-reviewedBioengineering