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

Figure 7. Systemic AAV-mxABE delivery sustains dystrophin restoration after 6 months.

Figure 7

(A) Heatmap of base editing rate in heart, DI, and TA muscle 6 months after treatment with AAV-mxABE (n = 4). (B) mxABE expression level in heart, DI, and TA 6 months after systemic injection (n = 4). (C) Western blot analysis of dystrophin (MilliporeSigma, D8168) restoration in different muscle tissues 6 months after treatment. (D) Quantification of Western blot results in C. (E) Histological immunostaining analysis of Dys+ (Abcam, ab15277) muscle area in heart, DI, and TA 6 months after treatment (n = 3). Scale bar: 200 μm. (F) Quantification of immunostaining results in E (n = 4). Dots and bars represent biological replicates and are mean ± SEM. Unpaired Student’s t test. Significance is indicated by an asterisk.