Recent studies have shown that antisense oligonucleotides and RNAi approaches targeting the PMP22 transcript are effective in rodent models of CMT1A.29,30 As cellular studies have already shown that overexpression of miR-29a in dermal fibroblasts derived from CMT1A patients downregulates human PMP22 expression,23 this may prove to be an attractive therapeutic target in a dosage-sensitive disease. This evidence concerns the gene PMP22 and Charcot-Marie-Tooth disease type 1A.