2 o methyl rna oligomers

Mdx (C57BL/10ScSn-Dmd^mdx/J) mice and wild type C57BL/10J mice were obtained from The Jackson Laboratory. The phosphorodiamidate morpholino modified antisense oligomer against the exon 23 of DMD gene (PPMO23) was synthesized and conjugated to the cell-penetrating peptide (RXRRBRRXRRBRXB) (Gene-Tools, LLC.). Male mdx mice were subjected to PPMO23 (15 mg/kg, i.v., biweekly), from 4 week of age, and combined with AGR-H19 (10 mg/kg s.q., every three days) or Nifenazone (10 mg/kg i.p. daily), and the experiment was terminated at 24 week of age. Male wild type C57BL/10J mice of same age were included as control group. The human antisense oligonucleotide (AON) against the exon 44 (h44AON1) of the human DMD gene was synthesized using phosphorothioated 2’O-Methyl RNA oligomers (Biosynthesis Inc.) and delivered to iPS-SkMCs/-CMs, scramble RNA was delivered as the negative control. H19 mimics and Nifenazone was used to treat the iPS-SkMCs/-CMs in combined with h44AON1, vehicle was used as the negative control. All cells were harvested 7 days post-treatment. Oligonucleotide sequences are included in the Supplementary Table 9.