Fig. 3 Correction of Dmd exon 44 deletion in mice by intramuscular AAV9 delivery of gene editing components. Correction of Dmd exon 44 deletion in mice.

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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. 3 Correction of Dmd exon 44 deletion in mice by intramuscular AAV9 delivery of gene editing components. Correction of Dmd exon 44 deletion in mice by intramuscular AAV9 delivery of gene editing components. (A) RT-PCR analysis of TA muscles from WT and ΔEx44 mice 3 weeks after intramuscular injection of gene editing components carried by AAV9. Lower dystrophin bands (179 bp) indicate skipping of exon 45. (B) Pie chart showing percentage of events detected at exon 45 after AAV-Cas9 and AAV-G6 treatment using RT-PCR sequence analysis of TOPO-TA generated clones. RT-PCR products were divided into four groups: NE, not edited; SK, exon 45 skipped; RF, reframed; and OF, out of frame. Data are represented as mean ± SEM (n = 3). (C) Sequences of RT-PCR products of WT, ΔEx44, and corrected ΔEx44 mice. In-frame sequences are shown in blue, including WT and exon 45–skipped sequences. Reframed sequence is shown in green, and out-of-frame sequence is shown in red. (D) Western blot analysis shows restoration of dystrophin expression in the TA muscle and heart of ΔEx44 mice. Vinculin is the loading control. (E) Quantification of the Western blot analysis in the TA muscle. Relative dystrophin intensity was calibrated with vinculin internal control. Data are represented as mean ± SEM. Unpaired Student’s t test was performed. *P < 0.005 (n = 3). (F) Immunostaining shows restoration of dystrophin in the TA muscle of ΔEx44 mice 3 weeks after intramuscular injection of gene editing components carried by AAV9. Dystrophin is shown in red. Nuclei are marked by DAPI stain in blue. Scale bar, 100 μm (n = 3). (G) H&E staining of the TA and heart in WT, ΔEx44, and corrected ΔEx44 mice. Inset boxes indicate areas of magnification shown below. Scale bars, 50 μm (n = 3). 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).