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Small molecule-based targeting of r(CUG)exp repeats in DM1. (a) CTG expansion in the DMPK locus results in the transcription of r(CUG)exp repeats that then sequester MBNL proteins, leading to some of the characteristic phenotypes in DM1. Abbreviations: DMPK, DM1 protein kinase; MBNL, Muscle-blind protein 1. (b) Representative small molecule inhibitors of steps in DM1 repeat pathogenesis.
