Recent advances in the treatment of Charcot-Marie-Tooth neuropathies.

Charcot-Marie-Tooth (CMT) neuropathies are one of the most common neuromuscular disorders. However, despite the identification of more than one-hundred causative genes, therapeutic options are still missing. The generation of authentic animal models and the increasing insights in the understanding of disease mechanisms, in addition to extraordinary developments in gene and molecular therapies, are quickly changing this scenario, and several strategies are currently being translated, or are getting close to, clinical trials. Here we provide an overview of the most recent advances for the therapy of CMT at both the preclinical and clinical level. For clarity, we have grouped the approaches in three different categories: gene therapy based on viral mediated delivery, molecular therapies based on alternative delivery systems, and pharmacological therapies.