Fig. 3.

Evaluation of the therapeutic effectiveness of the rAAV-microdystrophin treatment of mdx^5cv degeneration and regeneration reporter strains. (A and B) Representative BLI images of (A) degeneration reporter and (B) regeneration reporter strains imaged at 9 mo after the gene therapy treatment. Mice are wild type (Left), control (saline injected) mdx^5cv (Middle), and mdx^5cv where the left hindlimb (white arrow) had been injected with rAAV6-microdystrophin virus (Right). Tamoxifen treatments were done at 2 mo of age; control and rAAV injections were performed 2 wk after last dose of tamoxifen. (C and D) Quantitation of BLI signals at 3, 6, and 9 mo after rAAV injection from groups of mice illustrated in A and B. Data represent the mean ± SEM; n = 3–5; *P < 0.05; ***P < 0.0001. (E and F) Immunostaining for luciferase (red), dystrophin (green), and laminin (cyan) of cryosections from tibialis anterior muscles of tamoxifen-treated degeneration (E) and regeneration (F) reporter mice, 9 mo after hindlimb injections with saline or rAAV6-microdystrophin. 4,6-diamidino-2-phenylindole appears as blue. (Scale bar: 50 µm.)