Effective Dystrophin Restoration by a Novel Muscle-Homing Peptide-Morpholino Conjugate in Dystrophin-Deficient mdx Mice

(Mol Ther. 22, 1333–1341; July 2014) The authors have discovered that the original article has errors in Figures 4, S1, and S2 that were introduced when a graduate student used the wrong shared image folder when putting together the figures. In the original Figure 4a, which shows expression of dystrophin-positive fibers in body-wide muscles, control images for wild-type C57 and untreated mdx mice were repeatedly used instead of the right images corresponding to the mice used for this manuscript. The control images have been replaced and a corrected Figure 4a now appears below. In the original Figure 4d, which shows expression of dystrophin-associated protein complex in quadriceps, control images for wild-type C57 and untreated mdx mice were repeatedly used instead of the right images corresponding to the mice used for this manuscript. The control images have been replaced and a corrected Figure 4d now appears below. In the original Figure S1a, which shows expression of dystrophin-associated protein complex in quadriceps, control images for wild-type C57 and untreated mdx mice were repeatedly used instead of the right images corresponding to the mice used for this manuscript. The control images have been replaced and a corrected Figure S1a now appears below. In the original Figures S2a and S2b, a note should be added to the figure legend: the representative control images for C57 and mdx mice shown in Figure 5b are repeated in Figure S2a, and the representative control images for C57 and mdx mice shown in Figure 5a are repeated in Figure S2b. The corrected legend appears below. These corrections do not change the conclusions of the paper. The authors apologize for the errors and any confusion they may have caused.Figure 4dFunctional and phenotypic correction in mdx mice following treatment with M12–PMO conjugates at a single dose of 75 mg/kg intravenouslyView Large Image Figure ViewerDownload Hi-res image Download (PPT)Figure S1aFunctional evaluation of skeletal muscles following treatment with M12–PMO conjugates with 3 weekly injections at the dose of 25 mg/kg in mdx miceView Large Image Figure ViewerDownload Hi-res image Download (PPT) Figure S2. Histological and immunological measurement of potential toxicity in mdx mice treated with the M12–PMO conjugate with 3 weekly injections at 25 mg/kgThe representative control images for C57 and mdx mice shown in Figure 5b are repeated in Figure S2a. The representative control images for C57 and mdx mice shown in Figure 5a are repeated in Figure S2b. Effective Dystrophin Restoration by a Novel Muscle-Homing Peptide–Morpholino Conjugate in Dystrophin-Deficient mdx MiceGao et al.Molecular TherapyJuly, 2014In BriefAntisense oligonucleotide (AO)–mediated splice correction therapy for Duchenne muscular dystrophy has shown huge promise from recent phase 2b clinical trials, however high doses and costs are required and targeted delivery can lower both of these. We have previously demonstrated the feasibility of targeted delivery of AOs by conjugating a chimeric peptide, consisting of a muscle-specific peptide and a cell-penetrating peptide, to AOs in mdx mice. Although increased uptake in muscle was observed, the majority of peptide–AO conjugate was found in the liver. Full-Text PDF Open Archive