NEWS
Research group demonstrates the potential of base editing for HMSN-P
A research group including the Center for iPS Cell Research and Application (CiRA), RIKEN, Tokushima University, and Kobe University reported proof of concept for base editing designed to correct the TFG P285L variant responsible for HMSN-P. The work used both a mouse model and patient-derived neuromuscular organoids.
These findings are at the basic and preclinical research stage and do not represent a treatment currently available to patients.