Fig. 4 Systemic AAV9 delivery of gene editing components to ΔEx44 mice rescues dystrophin expression. Systemic AAV9 delivery of gene editing components.

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Fig. 4. Intramuscular injection of AAV9-Cas9/sgRNA-51 corrects dystrophin expression. Intramuscular injection of AAV9-Cas9/sgRNA-51 corrects dystrophin.

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Fig. 4 Systemic AAV9 delivery of gene editing components to ΔEx44 mice rescues dystrophin expression. Systemic AAV9 delivery of gene editing components to ΔEx44 mice rescues dystrophin expression. (A) Immunostaining shows restoration of dystrophin in the TA, triceps, diaphragm, and heart of ΔEx44 mice 4 weeks after systemic delivery of AAV-Cas9 and AAV-G6 at the indicated ratios. Dystrophin is shown in red. Nuclei are marked by DAPI stain in blue. Scale bar, 100 μm (B) Western blot analysis shows restoration of dystrophin expression in the TA, triceps, diaphragm, and heart of ΔEx44 mice 4 weeks after systemic delivery of AAV-Cas9 and AAV-G6 at the indicated ratios. Vinculin the loading control (n = 4). (C) Maximal tetanic force of the EDL muscles in WT (blue), ΔEx44 DMD (red), and corrected ΔEx44 DMD (green) mice 4 weeks after systemic delivery of AAV-Cas9 and AAV-sgRNA at 1:5 and 1:10 ratios. P < 0.005 (n = 6). (D) Specific force (mN/mm2) of the EDL muscles in WT (blue), ΔEx44 DMD (red), and corrected ΔEx44 DMD (green) mice 4 weeks after systemic delivery of AAV-Cas9 and AAV-sgRNA at 1:5 and 1:10 ratios. Data are represented as mean ± SEM. One-way ANOVA was performed, followed by Newman-Keuls post hoc test. **P < 0.001 (n = 6). Yi-Li Min et al. Sci Adv 2019;5:eaav4324 Copyright © 2019 The Authors, some rights reserved; exclusive licensee American Association for the Advancement of Science. No claim to original U.S. Government Works. Distributed under a Creative Commons Attribution NonCommercial License 4.0 (CC BY-NC).