Fig. 3: Restoration of FL-dystrophin expression in mdx^4cv mice following systemic MyoAAV4A delivery of Dys-N1/M3/C6.

All animal experiments were performed in WT and mdx^4cv male mice with the C57BL/6J genetic background. a The diagram depicting retro-orbital injection of MyoAAV4A carrying Dys-N1/M3/C6, the three MyoAAV4A constructs, and their respective doses administered. b Immunofluorescence images of GA muscle sections from WT and mdx^4cv mice treated with or without AAV (10 weeks of age, n = 5 per group), stained with three different anti-dystrophin antibodies recognizing N, M, and C fragments, respectively. Scale bar: 50 µm. c Quantification of dystrophin-positive muscle fibers in GA muscles of 10-week-old mdx^4cv mice treated with or without AAV (n = 5 per group, two-tailed unpaired Student’s t-test). d Western blotting analysis of dystrophin expression in GA muscles from WT and mdx^4cv mice treated with or without AAV (10 weeks of age, n = 3 per group). e Immunofluorescence staining of GA muscle sections from WT and mdx^4cv mice treated with or without AAV (10 weeks of age, n = 5 per group) using the antibodies against the DGC components including α-SG, β-SG, α-DG, β-DG, nNOS, and α-DB. Scale bar: 50 µm. Data were mean ± SEM. Source data are provided as a Source Data file. a created with BioRender.com released under a Creative Commons Attribution-NonCommercial-NoDerivs 4.0 International license (https://creativecommons.org/licenses/by-nc-nd/4.0/deed.en).