In vivo correction of cystic fibrosis mediated by PNA nanoparticles
A. S. Piotrowski-Daspit, C. Barone, C.-Y. Lin, Y. Deng, D. Wu, T. C. Binns, E. Xu, A. S. Ricciardi, R. Putman, R. Nguyen, A. Gupta, R. Fan, P. Glazer, W. M. Saltzman, 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.
bioRxiv · Jan 28, 2022Bioengineering