2'-O-[2-[(N,N-dimethylamino)oxy]ethyl]-modified oligonucleotides inhibit expression of mRNA in vitro and in vivo

Nucleic Acids Res. 2004 Feb 3;32(2):828-33. doi: 10.1093/nar/gkh220. Print 2004.

Abstract

Synthesis and antisense activity of oligonucleotides modified with 2'-O-[2-[(N,N-dimethylamino)oxy] ethyl] (2'-O-DMAOE) are described. The 2'-O-DMAOE-modified oligonucleotides showed superior metabolic stability in mice. The phosphorothioate oligonucleotide 'gapmers', with 2'-O-DMAOE- modified nucleoside residues at the ends and 2'-deoxy nucleosides residues in the central region, showed dose-dependent inhibition of mRNA expression in cell culture for two targets. 'Gapmer' oligonucleotides have one or two 2'-O-modified regions and a 2'-deoxyoligonucleotide phosphorothioate region that allows RNase H digestion of target mRNA. To determine the in vivo potency and efficacy, BalbC mice were treated with 2'-O-DMAOE gapmers and a dose-dependent reduction in the targeted C-raf mRNA expression was observed. Oligonucleotides with 2'-O-DMAOE modifications throughout the sequences reduced the intercellular adhesion molecule-1 (ICAM-1) protein expression very efficiently in HUVEC cells with an IC(50) of 1.8 nM. The inhibition of ICAM-1 protein expression by these uniformly modified 2'-O-DMAOE oligonucleotides may be due to selective interference with the formation of the translational initiation complex. These results demonstrate that 2'-O-DMAOE- modified oligonucleotides are useful for antisense-based therapeutics when either RNase H-dependent or RNase H-independent target reduction mechanisms are employed.

MeSH terms

  • Animals
  • Cell Line
  • Chromatography, High Pressure Liquid
  • Down-Regulation / drug effects*
  • Female
  • Humans
  • Intercellular Adhesion Molecule-1 / biosynthesis
  • Intercellular Adhesion Molecule-1 / genetics
  • Liver / drug effects
  • Liver / metabolism
  • Mass Spectrometry
  • Mice
  • Mice, Inbred BALB C
  • Molecular Structure
  • Oligonucleotides, Antisense / chemical synthesis
  • Oligonucleotides, Antisense / chemistry*
  • Oligonucleotides, Antisense / metabolism
  • Oligonucleotides, Antisense / pharmacology*
  • Peptide Chain Initiation, Translational / drug effects
  • Protein Biosynthesis / drug effects*
  • Protein Biosynthesis / genetics
  • Protein Kinase C / genetics
  • Protein Kinase C-alpha
  • Proto-Oncogene Proteins c-raf / genetics
  • RNA Stability
  • RNA, Messenger / genetics
  • RNA, Messenger / metabolism*
  • Ribonuclease H / metabolism

Substances

  • Oligonucleotides, Antisense
  • RNA, Messenger
  • Intercellular Adhesion Molecule-1
  • Proto-Oncogene Proteins c-raf
  • PRKCA protein, human
  • Prkca protein, mouse
  • Protein Kinase C
  • Protein Kinase C-alpha
  • Ribonuclease H