Fig. 7: Dose-dependent inhibition of expression of genes associated with heart failure and fibrosis. | Nature Communications

Fig. 7: Dose-dependent inhibition of expression of genes associated with heart failure and fibrosis.

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

Fig. 7

Transcriptional analysis of cardiac tissue from homozygous mice dosed retro-orbitally IV with vehicle or the indicated doses (vg/kg) of AAV9:mMybpc3 at two weeks of age and WT littermates for heart failure markers a Nppb, b Nppa, and c Myh7. Dose-dependent decreases in fibrotic marker expression in treated animals as assessed for d Col3a1, e Col4a1, and f Postn. Significant correlation between ejection fraction and MYBPC3 restoration as analyzed by transgene g RNA expression, h protein expression, and i cardiac transduction. Values are based on a simple linear regression model in GraphPad Prism. Analysis was performed 14 weeks post-injection. WT (n = 9; 5 M/4 F), Mybpc3-/- Veh (n = 10; 5 M/5 F), Mybpc3-/- 1E13 vg/kg (n = 7; 4 M/3 F), and Mybpc3-/- 3E13 vg/kg (n = 7; 3 M/4 F); preserved protein lysate unavailable for one 3E13 vg/kg animal. Data are shown as means ± SEM. P-value per one-way ANOVA with Tukey’s multiple comparisons test. Source data are provided as a Source Data file.

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