Fig. 3: AAV9:mMybpc3 and TN-201 improved hypertrophy, cardiac dysfunction, and premature lethality of Mybpc3-/- mice. | Nature Communications

Fig. 3: AAV9:mMybpc3 and TN-201 improved hypertrophy, cardiac dysfunction, and premature lethality of Mybpc3-/- mice.

From: AAV9-mediated MYBPC3 gene therapy with optimized expression cassette enhances cardiac function and survival in MYBPC3 cardiomyopathy models

Fig. 3

a LV mass normalized to body weight progression over time and b at 31 weeks post-delivery. c EF progression and d EF at 31 weeks post-delivery. e Left ventricular posterior wall thickness in diastole (LVPW;d) and f QT interval at 31 weeks post-delivery. g Kaplan–Meier survival curve with animals followed out until 20 months of age. Median survival for Mybpc3-/- vehicle animals was 14 months, and all animals died by 16 months of age. For TN-201-treated animals, median survival was 16 months (i.e. 2 months lifespan extension from vehicle), and all animals died by 19 months of age. AAV9:mMybpc3 treatment extended lifespan, with only one animal euthanized due to skin lesions not related to heart failure by 20 months. WT (n = 12; 5 M/7 F), Mybpc3-/- Veh (n = 11; 5 M/6 F; 10 at 31 weeks), Mybpc3-/- AAV9:mMybpc3 (n = 12; 6 M/6 F) and Mybpc3-/- TN-201 (n = 9; 4 M/5 F). Data are shown as means ± SEM. WT mice were significantly different from all groups for all parameters, with the exception of AAV9:mMybpc3-treated animals for QT interval. P-value per one-way ANOVA with Tukey’s multiple comparisons test. Mantel–Cox test used for Kaplan–Meier survival curve analysis. Source data are provided as a Source Data file.

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