Effect of BIX01294 on +1CGG RAN translation in HEK293 cells. NLuc levels from HEK293 cells transfected with AUG-NLuc or +1CGGx100-NLuc–expressing plasmids and treated with BIX01294 at the indicated doses for 24 h. Luminescence values are expressed relative to DMSO-treated cells (0 μm). Graphs represent mean ± S.D., with each n represented by a dot. n = 12 for 0 and 50 μm doses, n = 9 for 12.5 and 25 μm doses, and n = 6 for 37.5 μm, from 2–4 independent experiments with n = 3 each. *, p < 0.0001, two-way ANOVA with Sidak's multiple-comparison test.