Fig. 3
Correction of dystrophin expression by systemic delivery of AAV9-encoded gene editing components. a ΔEx50-Dmd-Luc mice were injected intraperitoneally with AAV9-Cas9 and AAV9-sgRNA-51 and analyzed by bioluminescence. Control mice were injected with saline. b Bioluminescence imaging of WT-Dmd-Luc mice and ΔEx50-Dmd-Luc mice injected with AAV9-Cas9 and AAV9-sgRNA. c Bioluminescent imaging (BLI) measurements of WT-Dmd-Luc mice, control ΔEx50-Dmd-Luc mice and ΔEx50-Dmd-Luc mice injected with AAV9-Cas9 and AAV9-sgRNA. d Dystrophin immunohistochemistry of diaphragm, heart, tibialis anterior, and triceps muscles 10 weeks after systemic injection with AAV9-Cas9 and AAV9-sgRNA. Data are represented as mean ± SEM. n = 4. (***P < 0.0005). Scale bar: 50 μm
