Charcot-Marie Tooth disease: A 100% French RNA-based therapeutic innovation

© Liliane Massade, Diseases and hormones of the nervous system (INSERM/Université Paris-Saclay) - O    Charcot-Marie Tooth disease causes progressive paralysis of the legs and hands. No treatment is available today. o    After more than a decade of research, a French team has developed an RNA-based therapy that allows model mice to regain their mobility. Charcot-Marie Tooth disease is the most common hereditary neurological disease in the world. It affects the peripheral nerves and causes progressive paralysis of the legs and hands. No treatment is currently available to fight this disease, which is due to the overexpression of a specific protein. Scientists from the CNRS, INSERM, the AP-HP and the Paris-Saclay and Paris universities have developed a therapy based on degrading the coding RNA for this protein in mice.