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). The gene discussed is MBNL1; the disease is Myotonia.