Figure 7.

Analysis of exon 23 skipping in gastrocnemius, quadriceps, diaphragm, and heart muscles of mdx mice treated with varying doses of αTfR1-MCC-pmoEx23 DAR3.7. mdx mice were treated via iv injection on Day 0 with αmTfR1-MCC-pmoEx23 DAR3.7 (10, 30, or 60 mg/kg PMO doses) or 3 doses of 10 mg/kg on Days 0, 7, and 14. Control groups included mdx and B10 WT mice treated with PBS via IV injection on Day 0. Mice (n = 4 per group/time point, error bars represent SEM) receiving a single dose were euthanized for tissue collection on Days 7, 14, 28, 56, and 84 post dose. Mice receiving 3 × 10 mg/kg doses, were euthanized for tissue collection on Days 28, 56, and 84 postfirst dose. Exon skipping was assessed by ddPCR.