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 100 causative genes, therapeutic options are still missing. The generation of authentic animal models and the increasing insights into 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 levels. 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.

Automated summary

Charcot-Marie-Tooth (CMT) neuropathies, despite a large number of identified causative genes, lack effective therapeutic options. However, recent advances in understanding disease mechanisms, animal modeling, and gene and molecular therapies have greatly improved the prospects of treating CMT, with several strategies progressing towards clinical trials.