BioengineeringbioRxivPreprint
Engineered CRISPR-Base Editors as a Permanent Treatment for Familial Dysautonomia
Shuqi Yun, Anil K. Chekuri, Jennifer Art…·Christiano R. R. Alves
This paper develops a CRISPR base editing strategy to correct the FD-causing mutation in human cells and mice, achieving up to 70% on-target correction and functional restoration.