Figure 1: Identification of small molecules that inhibit the formation of the r(CUG)12–MBNL1 complex.
From: Induction and reversal of myotonic dystrophy type 1 pre-mRNA splicing defects by small molecules

(a top) The biochemical outcome of a small molecule that targets r(CUG)exp would be the improvement of DM1-associated pre-mRNA splicing defects. (a bottom) The biochemical outcome of a small molecule that targets MBNL1 would be the induction of DM1-associated pre-mRNA splicing defects. (b) The small molecules that inhibit formation of the r(CUG)12–MBNL1 complex as identified by screening of the NIH’s MLPCN library.