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. Author manuscript; available in PMC: 2022 Jun 24.
Published in final edited form as: J Med Chem. 2021 Jun 8;64(12):8474–8485. doi: 10.1021/acs.jmedchem.1c00414

Figure 2.

Figure 2.

Small molecules that inhibit r(CCUG)exp–MBNL1 complex formation in vitro. (A) Characteristics of an RNA-focused small molecule library compared to characteristics of known drugs in DrugBank. (B) IC50 values of hit compounds from the RNA-focused library screen for disrupting the r(CCUG)12–MBNL1 complex (n = 2). Molecules in red have IC50s < 50 μM. (C) Chemical structures of compounds with IC50s < 50 μM. (D) Rescue of the IR exon 11 splicing defect by 3, 4, 5, and 7 in DM2 patient-derived fibroblasts (n = 2). Error bars represent standard deviation (SD) for all panels.