Ascorbic acid treatment corrects the phenotype of a mouse model of Charcot-Marie-Tooth disease Edith Passage1, 5, Jean Chrétien Norreel1, 2, 5, Pauline Noack-Fraissignes1, Véronique Sanguedolce1, Josette Pizant1, Xavier Thirion3, Andrée Robaglia-Schlupp1, Jean François Pellissier4 & Michel Fontés1 Charcot-Marie-Tooth disease (CMT) is the most common hereditary peripheral neuropathy, affecting 1 in 2,500 people. The only treatment currently available is rehabilitation or corrective surgery. The most frequent form of the disease, CMT-1A, involves abnormal myelination of the peripheral nerves. Here we used a mouse model of CMT-1A to test the ability of ascorbic acid, a known promoter of myelination, to correct the CMT-1A phenotype. Ascorbic acid treatment resulted in substantial amelioration of the CMT-1A phenotype, and reduced the expression of PMP22 to a level below what is necessary to induce the disease phenotype. As ascorbic acid has already been approved by the FDA for other clinical indications, it offers an immediate therapeutic possibility for patients with the disease. 1. Institut National de la Santé et de la Recherche Médicale UMR491, IPHM, Faculté de Médecine de la Timone, 27 Bd. J. Moulin, 13385 Marseille Cedex 5, France. 2. UPR Centre National de la Recherche Scientifique 9011, 31 Chemin J. Aiguier, 13402 Marseille Cedex 20, France. 3. Laboratoire de Santé Publique, EA 3279, Faculté de Médecine de la Timone, 27 Bd. J. Moulin, 13385 Marseille Cedex 5, France. 4. Service d'Anatomie Pathologique, CHU Timone Adulte, 264 Rue St. Pierre, 13385 Marseille Cedex 5, France. 5. These authors contributed equally to this work. Correspondence should be addressed to M Fontés. e-mail: Michel.Fontes@medecine.univ-mrs.fr Nature Medicine, Published online: 21 March 2004, doi:10.1038/nm1023 April 2004 Volume 10 Number 4 pp 396 - 401 Remember we are NOT Doctors and have NO medical training. This site is like an Encylopedia - there are many pages, many links on many topics. Support our work with any size DONATION - see left side of any page - for how to donate. You can help raise awareness of CAM.