Figure 2.
Systemic delivery of scAAV9/cre into Mecp2stop/y mice rescues abnormal respiration and open field motor activities. A, Experimental paradigm. Mice were analyzed 4–5 weeks after injection. B, Kaplan-Meier survival curve shows significant extension in longevity in scAAV9/cre mice. ***p < 0.001, *p < 0.05, log-rank test. C, Observational scores show stabilization in Mecp2stop/y mice injected with scAAV9/cre (n = 7), scAAV9/Control (n = 5), and noninjected Mecp2+/y mice (n = 12). D, E, Open field activity in scAAV9/cre (n = 3), scAAV9/Control (n = 3), and Mecp2+/y (n = 4) mice. F, Representative plethysmographic recordings from an age-matched Mecp2+/y mouse (top), Mecp2Stop/y mouse before (middle), and 5 weeks post scAAV9/cre injection (bottom). Scale shows time and tidal volume. G, Respiratory pattern returns to normal in scAAV9/cre (n = 6), scAAV9/Control (n = 3), and Mecp2+/y (n = 12) mice. In D, E, and G, *p < 0.05, **p < 0.01, ***p < 0.001, one-way ANOVA (Newman–Keuls multiple-comparison test). Data are means ± SEM.