Figure 2.

Functional efficacy of AAV.CAPN3 gene therapy by treadmill performance

(A and B) In the treadmill test, the LD (3 – 10¹² vg) and HD (6 × 10¹² vg) treatment cohorts showed significant improvements in (A) old and (B) young age groups compared with UT CAPN3 KO mice, reaching performance levels of age-matched WT mice. (C) Females of the HD cohort from the old group performed significantly better than the males of the same group. (D) Age at onset of treatment did not show a significant difference in treadmill outcomes. Each bar represents the mean ± SEM of 12–14 mice per CAPN3 KO cohort and 8–14 mice per age-matched WT mouse. ∗∗p < 0.01, ∗∗∗p < 0.001, ∗∗∗∗p < 0.0001, one-way ANOVA, Tukey’s multiple comparisons test for (A) and (B) and two-way ANOVA, Sidak’s multiple comparison test for (C) and (D).