Fig. 4: Additional validation of AAVMYO.

a Shown on top are whole-body images of luciferase expression in CB17-SCID mice 4 weeks after i.v. injection with the shown vectors. Shown underneath are images of luciferase expression in the indicated organs from the same mice, harvested 1 week later. b Shown are fold-changes of luciferase mRNA expression of each organ as determined by qRT-PCR in the AAVMYO cohort versus the AAV9 group. Depicted values are the average of the five CB17-SCID mice from panel a with SD; *p < 0.05, **p < 0.01, ***p < 0.001, and ****p < 0.0001 (unpaired two-tailed t test). c Comparison of µDys expression in quadriceps femoris sections (n = 4 replicates for all groups) and protein lysates of mdx mice 4 weeks after i.v. injection of 2 × 10¹¹ vg (for IHC) or 1 × 10¹² vg (for western blot) of a control vector (AAVMYO expressing Firefly luciferase), or of AAV9 or AAVMYO encoding µDys. d Staining of muscle fibers type I (BA-F8), type IIa (sc-71), type IIb (BF-F3), and GFP in quadriceps femoris sections of C57BL/6 mice (n = 4 replicates for all groups) injected with 5 × 10¹¹ vg of AAVMYO. Scale bars for c and d are 25 and 50 µm, respectively. Source data are available in the Source data file.