We performed siRNA-mediated silencing of MBNL1 or both MBNL1 and MBNL2 in differentiated human muscle cells to mimic the functional loss of MBNL proteins in DM1 and showed that the silencing of MBNL1 is sufficient to promote an exclusion of DMD exon 78 (Fig. 1e, right panel). The gene discussed is DMD; the disease is myotonic dystrophy type 1.