MFN2 and Charcot-Marie-Tooth disease type 2A1: To develop a therapeutic strategy for CMT2A, we investigated a combined approach of RNA interference and gene replacement therapy (RNAi/gene therapy) in which both MFN2 mutant and WT alleles are inhibited by a shRNA sequence, while the Myc-DDK-tagged WT MFN2 protein is restored leveraging the transfer of a Myc-DDK-tagged MFN2 cDNA engineered to be resistant to the shRNA (Fig. 1A).