Author Archives: Ana Pena, PhD

Gene Therapy for CMT4C Fares Well in Preliminary Tests in Mice

A gene therapy strategy to treat Charcot-Marie-Tooth disease 4 subtype C (CMT4C), targeted to correct the myelin-producing cells of the peripheral nervous system, showed positive results in a mouse model. The data provide proof-of-principle support for gene therapy to treat CMT4C and potentially other similar demyelinating inherited nerve diseases.