The GDAP1 gene is involved in several forms of CMT (including CMT 2K and CMT 4A). It encodes a mitochondrial protein.
After screening in vitro 1018 molecules marketed in the United States, researchers have shown that florfenicol, an antibiotic that activates the respiratory chain, prevents the onset of symptoms (poor motor coordination and overweight) in mice deficient in GDAP1, treated presymptomatically.
Administration of an antioxidant, MitoQ, has comparable benefits, suggesting that the antioxidant response is involved in pathological mechanisms.
