LNP Delivery of Prime Editors Enables Durable Correction of PAH R408W in Phenylketonuria Models
Stephanie Yazinski, Susanna Canali, Yang Xu, Xuling Zhu, James Nelson, Wenguo Gao, Zharko Daniloski, Wendy Leung-Owen, Felicity Winkler, Andrea Frassetto, Anna Simon, Jon N Penterman, Sean F. Maguire, Danielle Fuchs, James Rybarski, , Patrick F. Finn
This paper demonstrates the use of LNP-delivered prime editors to correct the PAH R408W mutation in PKU models, achieving ~50% correction efficiency.
bioRxiv · Oct 14, 2026 · PreprintPharmacology & Toxicology