Figure 3: Systemic delivery of AAVMYO split intein/Dys enhances muscle targeting at low doses.

a) Plots representing TA specific force development of adult WT or mdx^4cv mice treated intravenously with saline, AAV serotype 9, or AAVMYO at low (2×10¹³ vg/kg) or high (2×10¹⁴ vg/kg) dose for 12 weeks. b) Muscle forces measured following a 15%-lengthening contraction beyond the optimal L0. c) Circulating Creatine Kinase levels measured in serum samples after 12 weeks of AAV treatment. d) Protein expression of μDys, midi-Dys (ΔSR5–15), or full-length dystrophin in T.A muscle lysates of control (WT or mdx^4cv) or AAV-treated mice. For a and b n=5 mice for low dose AAV9 or AAVMYO μDys5, n= 6 mice per group other groups, while in c, n=4 per group was used. Data represent means±s.e.m. *p<0.05, **p<0.01, ***p<0.001 versus mdx^4cv mice treated with saline, and ^$p<0.05, ^{$ $}p<0.01, ^{$ $ $}p<0.001 versus WT mice (ANOVA test followed by Tukey’s post hoc). μDys: micro-dystrophin. m-Dys: midi-dystrophin. Ab: antibody. kDa: kilodalton. MW: molecular weight. vg: viral genome. Kg: Kilogram.