Several therapeutic approaches have been used for the treatment of DM1 muscular symptoms including: the use of antisense oligonucleotide [13], reduction of CUG transcript level [14], inhibition of MBNL1 sequestration [15–17], cleavage of CUG expanded repeats by CRISPR/CAS9 editing [18] or small molecules [19] and blocking the production of CUG repeats by genome modification [20]. The gene discussed is MBNL1; the disease is myotonic dystrophy type 1.