Therapeutic tools for inherited neuropathies

Inherited neuropathies are a genetically and phenotypically heterogenous group of disorders leading to sensory and motor dysfunction. For years, these neuropathies have been considered as non-treatable diseases, as no drug is able to induce nerve regrowth. Progress in molecular tools has changed this view and several neuropathies can now be efficiently treated. Some more will be treatable in the upcoming years. Basically, these new treatments can be divided into four categories, depending on the target: gene therapy; gene expression therapy; protein modification or replacement (enzyme replacement therapy, ERT); downstream therapies. In this short review, we will provide a few examples for each of them in the field of peripheral neuropathies.