Pharmacology & ToxicologybioRxivPreprint
LNP Delivery of Prime Editors Enables Durable Correction of PAH R408W in Phenylketonuria Models
Stephanie Yazinski, Susanna Canali, Yang Xu…·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.
DOI 10.64898/2026.10.06.753751