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. Intramuscular injection of AAV9-Cas9/sgRNA-51 corrects dystrophin expression. Intramuscular injection of AAV9-Cas9/sgRNA-51 corrects dystrophin expression. (A) Tibialis anterior muscles of ΔEx50 mice were injected with AAV9 vector encoding sgRNA-51 and Cas9 (see Fig. 2) and were analyzed 3 weeks later by immunostaining for dystrophin. WT control (WT-CTL) mice and ΔEx50 control mice (ΔEx50-CTL) were injected with AAV9-Cas9 alone without sgRNAs. Indicated are percentages of dystrophin-positive myofibers in ΔEx50 mice receiving intramuscular injections of AAV9-Cas9/sgRNA-51 (ΔEx50-AAV9-sgRNA-51) compared to WT-CTL. (B) H&E staining of tibialis anterior muscles. (C) Western blot analysis of dystrophin (DMD) and VCL expression in tibialis anterior muscles 3 weeks after intramuscular injection of AAV9-Cas9 control or AAV9-Cas9/sgRNA-51. (D) Quantification of dystrophin expression from Western blots after normalization to VCL. Asterisk indicates nonspecific immunoreactive bands. n = 5 for AAV9-sgRNA-51. Scale bars, 50 μm. Leonela Amoasii et al., Sci Transl Med 2017;9:eaan8081 Published by AAAS