Fig. 3.

Ligand 4 as an inhibitor of r(CUG)^exp–MBNL1 and d(CTG⋅CAG)^exp transcription. (A) Reduction of disease foci in DM1 patient-derived myoblasts upon the treatment of oligomer at 200 nM. Foci areas are indicated with white arrows. (Scale bars, 5 μm.) (B) Schematic description of splicing pattern of IR pre-mRNA and the result of splicing assay in a presence of oligomer 4. Full recovery of the IR splicing defect was obtained at 200 nM. P values were calculated against data points of the diseased sample. *P < 0.05. No significant differences between normal cells and each of all the treated samples (P > 0.05, Student’s t test, two-tailed). (C) A plot of normalized RNA transcript level against the oligomer 4 concentration. RNA transcript level was normalized to the level of a loading reference RNA, HIV fs RNA, for quantitative comparison between the loading samples. Oligomer 4 inhibited d(CTG)^exp transcription in a dose-dependent manner (IC₅₀ = 100 nM), but no significant inhibition was detected with the control DNA template throughout all the tested concentrations. Error bars indicate standard error of the mean from three independent experiments.