DMPK and myotonic dystrophy type 1: We analyzed the effects of ASOs targeting the expanded CUG tract of mutant DMPK transcripts that inhibit the deleterious sequestration of MBNL1 splicing factor and reduce the number of nuclear CUGexp-RNA aggregates, thereby correcting DM1 splicing defects in DM1 mouse models (Mulders et al., 2009; Wheeler et al., 2009).