Characterization of a humanized mouse model of Charcot-Marie-Tooth type 1A for the discovery of human PMP22-targeting drugs

Homo-humanized mice reflect various aspects of CMT1A characteristics in an <i>hPMP22</i> gene dosage-dependent manner. This model will help us better understand the relationship between <i>PMP22</i> reduction levels and the recovery of CMT1A-related phenotypes, contributing to the translation of preclinical findings into clinically relevant human treatments and dosing strategies.