Fig. 4

Expression of dystrophin and luciferase following systemic delivery of AAV9-encoded gene editing components. a Western blot analysis of dystrophin (DMD), luciferase (Luc), Cas9, and vinculin (VCL) in diaphragm, heart, triceps muscles, and tibialis anterior of WT-Dmd-Luc mice, control ΔEx50-Dmd-Luc mice and ΔEx50-Dmd-Luc mice injected with AAV9-Cas9 and AAV9-sgRNA. b Quantification of dystrophin and luciferase expression from blots after normalization to vinculin. Data are represented as mean ± SEM. n = 4