Fig. 4.
Functional tests on mdx mice injected systemically with AAV-U1#23. Controls (black), untreated mdx (blue), and AAV-U1#23 (red)-treated mdx mice. (A) Specific force of single fibers from the gastrocnemius (n = 120, mice n = 3 for each group) was significantly lower in mdx mice in comparison with controls (P ≤ 0.01) and recovered toward normal values in AAV-U1#23 (+30%, P ≤ 0.01). (B) Po/CSA of fibers (n = 150, mice n = 3 for each group) from the vastus of C57BL/6 and mdx and from small GFP-positive and GFP-negative bundles of AAV-U1#23-treated muscles. GFP-positive fibers from treated mice showed Po/CSA values significantly higher (P ≤ 0.001) than those obtained from GFP-negative fibers of treated mice and fibers of untreated mdx mice (P ≤ 0.001). (C) CKnac (units/liter) serum levels were monitored 3 and 6 weeks after AAV-U1#23 injections in parallel to C57BL/6 and mdx mice. (D) Untreated mdx mice showed a significant reduction (P ≤ 0.001) of the tolerance to treadmill exhaustion test compared with C57BL/6, whereas a partial but significant recovery was observed after AAV-U1#23 treatment (P ≤ 0.01). ∗, significantly different from the other groups; bullet, significantly different from C57BL/6 and AAV-U1#23 GFP-positive. Data are mean values ± SEM. Statistical analysis was assessed by ANOVA followed by the Student–Newman–Keuls test. P ≤ 0.05 was considered significant.