Fig. 3: Comparison of PMO or PMO/HDO effects on dystrophin expression. | Nature Communications

Fig. 3: Comparison of PMO or PMO/HDO effects on dystrophin expression.

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

Fig. 3

a Images of dystrophin immunostaining (red) in indicated muscles 2 weeks after the fifth PMO dose (100 mg/kg) or Chol-HDO at an equimolar dose to PMO (11.88 μmol/kg). Scale bar, 100 μm. b Quantification of dystrophin-expressing fibers (%) to the total number of fibers in the indicated tissues (n = 4 per group). c Myofiber cross-sectional area (CSA), and (d) percentage of centrally nucleated fibers (CNF) in quadriceps 2 weeks after five weekly injections of phosphate-buffered saline (PBS), PMOs, or HDOs (11.88 μmol/kg) (n = 4 per group). e Representative images of caveolin 3 immunostaining (red) in quadriceps femoris (QF) counterstained with DAPI (blue) to evaluate CNF and CSA. f Western blot showing robust dystrophin expression in the heart and QF from HDO-treated mice. Data are presented as mean ± S.E.M and were analyzed using one-way analysis of variance followed by Tukey’s tests (b–d). P-values are indicated. Source data are provided as a Source Data file.

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