Fig. 4: Systemic delivery of PMO/HDOs drastically improves the mdx phenotype. | Nature Communications

Fig. 4: Systemic delivery of PMO/HDOs drastically improves the mdx phenotype.

From: Heteroduplex oligonucleotide technology boosts oligonucleotide splice switching activity of morpholino oligomers in a Duchenne muscular dystrophy mouse model

Fig. 4

a Serum Creatine Kinase (CK) levels in mice injected once weekly for a total of 5 doses with PBS or HDOs (11.88 μmol/kg). Serum CK levels are reduced after treatment with Toc- or Chol-HDO, correlating with the levels of dystrophin restoration (n = 5-9 per group). b Forelimb grip test (n = 9–15 per group) and (c) treadmill test (n = 4–7 per group) performances evaluated in mdx mice injected once weekly for a total of 5 doses with PBS or HDOs (11.88 μmol/kg). ECG abnormalities observed in mdx are prevented in the treated mdx mice (d) QTc and (e) QRS duration (n = 6–11 per group). f Quantification of the heart fibrosis-stained regions in the left ventricle (n = 4 per group). Transverse sections revealing the level of the papillary muscles 8 weeks (2 months) after once weekly for a total of 5 doses of PBS or HDOs (11.88 μmol/kg). g Representative images of the heart after Masson’s trichrome staining in the left ventricle. Data are presented as mean ± S.E.M and were analyzed using one-way analysis of variance followed by Tukey’s Kramer tests (a–f). P-values are indicated. Source data are provided as a Source Data file.

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