Fig. 4: AAV9:mMybpc3 improves cardiac function at a dose as low as 1E13 vg/kg.

Dose-dependent improvement compared to vehicle-treated Mybpc3-/- mice in a LV mass normalized to body weight, b EF, and c QT interval at 31 weeks post-delivery. d No group differences in body weight at 31 weeks post-delivery. Data are shown as means ± SEM. WT mice were significantly different from all groups for all echocardiographic parameters, with the exception of 3E13 vg/kg and 1E14 vg/kg AAV9:mMybpc3-treated animals for QT interval. WT (n = 7; 4 M/3 F), Mybpc3-/- Veh (n = 7; 4 M/3 F), Mybpc3-/- 1E13 vg/kg (n = 4; 3 M/1 F), Mybpc3-/- 3E13 vg/kg (n = 6; 4 M/2 F), and Mybpc3-/- 1E14 vg/kg (n = 6; 4 M/2 F). P-value per one-way ANOVA with Tukey’s multiple comparisons test. Source data are provided as a Source Data file.