Figure 2.
Life-Long AAV-Mediated CRISPR Genome Editing Restored Dystrophin Expression in mdx Heart
(A) Representative immunofluorescence images of heart sections stained with the antibodies against dystrophin (green). Scale bar, 500 μm. (B) Western blot analysis of dystrophin expression in heart lysates from 19-month-old WT and mdx mice treated with or without rAAV-CRISPR (1 × 1012 vg, i.p. at day 3). Gapdh was used as a gel loading control. (C) Quantification of dystrophin-positive cardiomyocytes on immunofluorescence stained sections. (D) Densitometry quantification of relative expression of total dystrophin on western blot. Error bars indicate mean ± SEM.