Figure 4.

AAV9-IGHMBP2 repairs muscle pathology in gastrocnemius, quadriceps, and diaphragm. (a,e) Verhoeff-van Gieson-stained cross sections of gastrocnemius (a) and quadriceps (e) muscles of low-dose-treated males were examined at 8 weeks postinjection under 10× (top) and 40× (bottom) magnifications and compared with those of age-matched untreated nmd and “Het” (n = 4 for each group). (h) Verhoeff-van Gieson-stained longitudinal (top) and cross sections (bottom) of diaphragm of low-dose-treated males were examined at 8 weeks postinjection under 40× magnification and compared with those of age-matched untreated nmd and “Het” (n = 4 for each group). Untreated nmd mice demonstrate significant reduction in the muscle fiber area, replacement of muscles by adipose tissue (asterisks), centralized nuclei (arrow), and interstitial fibrosis (pink) while all of these defects have been significantly rescued in treated nmd mice (a,e,h). (b) The ratio of gastrocnemius weight to total body weight is increased in treated mice compared to untreated (one-way analysis of variance (ANOVA) P < 0.0001). (c,f,i) The area size of muscle fiber in treated nmd is significantly increased compared to untreated (one-way ANOVA P < 0.0001). (d,g,j) The intensity of interstitial/periarterial fibrosis is significantly decreased in the muscles of treated nmd compared to untreated (one-way ANOVA P < 0.0001). Scale bar = 200 m.