An increasing number of gene therapy products are being developed for Charcot-Marie-Tooth disease. This article provides an overview of the main approaches under study:
- a single phase I/II trial is underway in the United States in CMT 2S, involving 10 patients,
- twelve products are in preclinical development, for which the choice of vector and route of administration have yet to be optimised, and their safety has yet to be demonstrated.
While initial animal research suggests that gene therapy is a promising treatment for CMT, only an early injection could counteract the processes of demyelination and/or axonal loss. Combining gene therapy with other therapeutic approaches could optimise its benefits.
