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. 2023 Feb 1;133(3):e162809. doi: 10.1172/JCI162809

Figure 6. Restored dystrophin expression and muscle function 6 weeks after systemic mxABE treatment.

Figure 6

(A) Base editing efficiency in heart, DI, and TA muscle 6 weeks after systemic treatment with mxABE. Unpaired Student’s t test (n = 4). (B) Western blot analysis of dystrophin (MilliporeSigma, D8168) restoration in heart, DI, and TA. (C) Histological immunostaining analysis of Dys+ (Abcam, ab15277) muscle area in heart, DI, and TA after treatment. Scale bar: 200 μm. (D) Forelimb grip strength test results for WT, untreated, and M3- and C1-treated DMDE30mut mice. Unpaired Student’s t test (n = 4). (E) Exhausted strength test showed partial rescue of forelimb muscle function in mxABE-treated DMDE30mut mice. Different asterisks represent statistical significance (P < 0.01) in multiple-comparison test using ANOVA (n = 4). (F) Rotarod running test indicated functional muscle improvement in mxABE-treated DMDE30mut mice (n = 4). Dots and bars represent biological replicates and are mean ± SEM. Significance is indicated by asterisks.