Figure 6.

Impact of DAR on levels of (a) tissue concentration and exon skipping in (b) gastrocnemius, (c) quadriceps, (d) diaphragm, and (e) heart muscles. mdx mice were treated with a single IV injection of 10 mg/kg PMO dose for the DAR3.9 and DAR5.8 AOCs, and 12 mg/kg for the DAR9.7 AOCs. Mice (n = 4 per group, error bars represent SEM) were treated with different combinations of linkers (MCC or MC), DARs, and PMO conjugated to αmTfR1. Control groups included B10 WT mice and mdx mice treated with IV PBS. Tissues were harvested at 14 days post-treatment and exon skipping was assessed by ddPCR. Statistical analysis was performed using one-way ANOVA, with all groups compared against αmTfR1-MCC-pmoEx23 DAR4. *: P ≤ 0.05, **: P ≤ 0.01, ***: P ≤ 0.001; ****: P ≤ 0.0001. ANOVA, analysis of variance; ddPCR, droplet digital polymerase chain reaction; IV, intravenous; ns, not significant.