To test this therapeutic approach, we demonstrated that MBNL1 was overexpressed following intramuscular (i.m.)injection of rAAV2/1-mycMbnl1 virus, and this led to the reversal of myotonia and missplicing of DM1 target RNAs in the HSALR poly(CUG) model for DM1 (16). Here, MBNL1 is linked to myotonic dystrophy type 1.