Figure 3.

Small molecules inhibit CGG RAN translation in multiple reading frames. A, structures of compounds used in B–H. B–F, compounds identified from screen as RAN translation inhibitors were independently reassessed for their activity in RRL in vitro translation assays. The indicated compounds were added at increasing doses to 30% RRL reactions with AUG, +1CGGx100, or +2CGGx100-NLuc reporter mRNAs, and luminescence was measured relative to vehicle (DMSO) treatment. G and H, candidate compounds protoporphyrin IX and TMPyP4 were added at increasing concentrations to RRL reactions with AUG, +1CGGx100, or +2CGGx100-NLuc reporter mRNAs. Luminescence was measured relative to vehicle (DMSO) treatment. For all graphs, n = 3; error bars, S.D. *, p < 0.05, two-way ANOVA with Dunnett's multiple-comparison test.