The vast majority of experimental therapies are dedicated to the most common CMT subtypes, i.e., CMT1A (PMP22 gene duplication), CMTX1 (GJB1 gene point mutations), CMT1B (MPZ gene mutations) and CMT2A (MFN2 gene mutations), which, together, make up more than 90% of genetically confirmed CMT cases [18]. The gene discussed is GJB1; the disease is Charcot-Marie-Tooth disease.