BioengineeringbioRxiv
In vivo correction of cystic fibrosis mediated by PNA nanoparticles
A. S. Piotrowski-Daspit, C. Barone, C.-Y. Lin…·M. E. Egan
This study demonstrates systemic gene editing using PNA nanoparticles to correct the F508del CFTR mutation in mice, showing partial CFTR function restoration and tissue-specific correction without off-target effects.
DOI 10.1126/sciadv.abo0522