Figure 3.

Survival of Mmut^−/− mice, vector biodistribution, and MMUT protein expression after treatment with AAV44.9-CBA-Mmut

Mmut^−/− mice received an intrahepatic injection of 2 × 10¹¹ vg of AAV44.9-CBA-Mmut per pup at birth, and were sacrificed on day of life 60. (A) AAV44.9-CBA-Mmut treated Mmut^−/− mice had a significant increase in survival in comparison with untreated Mmut^−/− mice (∗∗∗p < 0.005, Log rank (Mantel-Cox) test). (B) Vector biodistribution of AAV44.9-CBA-Mmut in Mmut^−/− treated mice at day 60, n = 4 for all tissues studied (∗∗p < 0.01, two-way ANOVA). (C) MMUT hepatic protein expression in AAV44.9-CBA-Mmut treated Mmut^−/− mice (n = 3) determined by western blot analysis at day 60. Beta-actin served as a loading control. (D) Quantification of hepatic MMUT protein expression in (C) normalized to beta-actin. NS, not significant (one-way ANOVA with Kruskal-Wallis test); WT, wild type. Error bars = $\pm$ SEM.