Figure 4. AAV-mxABE robustly rescues dystrophin expression in TA 6 weeks after injection.

(A) Deep sequencing of in vivo RNA editing 6 weeks after i.m. injection with AAV9-E1, -C3, -C4, and -M2 constructs (n = 4). (B) Western blot analysis of dystrophin (MilliporeSigma, D8168) protein expression in TA muscles of WT and DMD^E30mut mice. Intramuscular injection of saline in the DMD^E30mut mice was the control. Vinculin was used as the loading control. (C) Quantification of dystrophin expression from Western blots after normalization to vinculin (n = 3). (D) Immunohistochemistry of dystrophin in TA muscles 6 weeks after i.m. injection with different AAV9 constructs. Dystrophin (Abcam, ab15277) is indicated in green. Scale bar: 200 μm. (E) Quantification of Dys⁺ fibers in cross sections of TA muscles (n = 4). Dots and bars represent biological replicates and are mean ± SEM. Unpaired 2-tailed Student’s t test. *P < 0.05, **P < 0.01 vs. control.