Base editing shown to correct HMSN-P mutation in preclinical study
A research group including CiRA, RIKEN, Tokushima University, and Kobe University demonstrated proof of concept for base editing targeting the TFG P285L variant responsible for HMSN-P. The study used mouse models and patient-derived neuromuscular organoids. These findings are at the basic and preclinical research stage and are not yet a treatment option.