Figure 1.

Life-Long AAV-Mediated CRISPR Genome Editing Improved Cardiomyopathy in mdx Mice

(A) Scheme illustrating the CRISPR/SaCas9-mediated genome-editing strategy to delete the exons 21–23 in order to restore the dystrophin reading frame. The sequences for the two gRNAs were shown with the PAM sequences italicized. (B) Representative H&E staining and Masson’s trichrome staining images of heart sections from 19-month-old WT and mdx mice treated with or without rAAV-CRISPR (1 × 10¹² vg, intraperitoneal [i.p.] at day 3). Scale bar, 50 μm. (C) Quantification of cardiac fibrotic area in trichrome stained heart sections. (D) Quantification of collagen content in Sirius red/fast green-stained heart sections. (E and F) Cardiac output (E) and stroke volume (F) were measured in 19-month-old WT and mdx mice by echocardiography. (G) Measurements of cardiac troponin I in the serum samples of mice at 19 months of age. *p < 0.05, **p < 0.01, ***p < 0.001. Error bars indicate mean ± SEM.