Fig. 1: Properties and biodistribution of PMOs and PMO/HDOs. | Nature Communications

Fig. 1: Properties and biodistribution of PMOs and PMO/HDOs.

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

Fig. 1

a Structure of phosphorodiamidate morpholino oligomers (PMOs) duplexed with lipid ligand (tocopherol (Toc) or cholesterol (Cho))-conjugated complementary strand. b Confirmation of annealing between PMOs and the complementary strand with lipid ligands electrophoresed on a 16% acrylamide gel. bp: base pairs (c, d) Pharmacokinetics of PMO after intravenous injection of a single 100 mg/kg (11.88 μmol/kg) PMO dose or molar equivalent of Toc-HDO or Chol-HDO. The hybridization-based ELISA shows the pharmacokinetic (c), and biodistribution (d) data in mdx mice (n = 4) injected with PMO, Toc-HDO or Chol-HDO. Data are presented as mean ± S.E.M. e Binding curves of PMO, lipid conjugated PMO/HDO with mouse albumin. Source data are provided as a Source Data file.

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