Figure 3. TG693 induces truncated dystrophin protein expression in patient-derived cells.

Immortalized DMD patient-derived cells were treated with increasing concentrations of TG693 for 2 d. (a) Semi-quantitative RT-PCR for dystrophin exon skipping. The uncropped image is provided in Supplementary Fig. S7. Data represent the means ± SD (n = 3). *p < 0.05. (b) Western blotting of dystrophin protein expression in TG693-treated cells using C-terminal-directed antibody. α-Tubulin was used as a loading control. Uncropped images have been provided in Supplementary Fig. S8. Data are representative of three independent experiments.