Fig. 4: Functional and histopathological improvement 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 Measurement of serum CK in 8-week-old mice (n = 14 WT, 12 mdx^4cv and 13 AAV-treated mdx^4cv mice). b Tetanic torque measurements of the posterior compartment muscles of the mice (n = 8 WT, 10 mdx^4cv and 11 AAV-treated mdx^4cv; 8−12 weeks of age). c H&E staining, immunofluorescence staining with anti-laminin α2/DAPI and Masson’s Trichrome staining images of GA muscle sections (n = 5 per group; 10 weeks of age). Scale bar: 50 µm. d Measurement of CNF in the GA muscles of 10-week-old mdx^4cv mice with or without AAV treatment (n = 5 each, two-tailed unpaired t-test). e Muscle fiber size distribution in GA muscles of the mice at 10 weeks of age (n =5 WT, 5 mdx^4cv, and 6 AAV-treated mdx^4cv). f Quantitative analysis of fibrotic area in the GA muscle sections of the mice at 10 weeks of age (n = 3 per group). Data were mean ± SEM. Statistical analyses were performed by one-way ANOVA with Tukey’s multiple comparisons test except otherwise specified. Source data are provided as a Source Data file.