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Unraveling the genetic and molecular mechanisms, as well as the cellular effects of CMT mutations, has facilitated the development of promising gene therapy approaches. Proposed gene therapy treatments for CMTs include virally or non\u2010virally mediated gene replacement, addition, silencing, modification, and editing of genetic material. For most CMT neuropathies, gene\u2010 and disease\u2010 and even mutation\u2010specific therapy approaches targeting the neuronal axon or myelinating Schwann cells may be needed, due to the diversity of underlying cellular and molecular\u2010genetic mechanisms. The efficiency of gene therapies to improve the disease phenotype has been tested mostly<jats:italic>in vitro<\/jats:italic>and<jats:italic>in vivo<\/jats:italic>rodent models that reproduce different molecular and pathological aspects of CMT neuropathies. 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