LNP Delivery of Prime Editors Enables Durable Correction of PAH R408W in Phenylketonuria Models
Phenylketonuria (PKU) is a monogenic metabolic disorder caused by mutations in the phenylalanine hydroxylase (PAH) gene, the most common of which is c.1222C>T (p.R408W). Current treatment options, including dietary restriction and enzyme substitution, can be effective but are often challenging for patients to maintain...
Stephanie Yazinski, Susanna Canali, Yang Xu et al.
· bioRxiv (Cold Spring Harbor... · 0 citations