Figure 2.

Long-Term, High-Dose Treatment of 24-Week-Old mdx Mice With PMO Results in Exon Skipping and Expression of Dystrophin

(A) Reverse transcriptase polymerase chain reaction performed on cardiac muscle ribonucleic acid from PMO-treated mdx mice demonstrating exon 23 skipping (Δ 23 687 bp) (indicated by asterisk) and age-matched untreated mdx and wt control mice. The 22/23 541 bp amplicon represents an out-of-frame transcript missing exons 22 and 23 and has been previously reported 37, 38. (B) Immunoblot performed on cardiac muscle from untreated wt (5%, 2%, and 1% dilutions), untreated mdx mice (undiluted), and PMO-treated mdx mice (undiluted) demonstrating presence of dystrophin (indicated by asterisk). (C) Immunostaining of heart cryosections from untreated wt and mdx mice and PMO-treated mdx mice demonstrating presence of dystrophin. Bars = 100 μm. PMO treatment: 40 mg/kg PMO 3× per week for 19 weeks; other abbreviations as in Figure 1.