Figure 3.

A single dose of FORCE–M23D, but not of unconjugated M23D, induces dose-dependent Dmd exon 23 skipping in skeletal and cardiac muscles of mdx mice. Five-week-old mdx mice were injected via tail vein with vehicle, 30 mg/kg unconjugated M23D, or 10 mg/kg or 30 mg/kg PMO-equivalents of FORCE–M23D and sacrificed at the indicated time points. Exon 23 skipping was measured by RT-PCR and capillary electrophoresis. Percent skipping was calculated as described in Materials and Methods. Data represent mean ± SD. * P < 0.05, ** P < 0.01, **** P < 0.0001. PMO, phosphorodiamidate morpholino oligomer; RT-PCR, reverse transcription polymerase chain reaction; WT, wild type.