Comparing in vitro and in vivo activity of 2'-O-[2-(methylamino)-2-oxoethyl]- and 2'-O-methoxyethyl-modified antisense oligonucleotides

J Med Chem. 2008 May 8;51(9):2766-76. doi: 10.1021/jm701537z. Epub 2008 Apr 10.

Abstract

A number of 2'- O-modified antisense oligonucleotides have been reported for their potential use in oligonucleotide-based therapeutics. To date, most of the in vivo data has been generated for 2'-O-MOE (2'-O-methoxyethyl)- and 2'-O-Me (2'-O-methyl)-modified ASOs (antisense oligonucleotides). We now report the synthesis and biological activity of another 2'-O-modification, namely 2'-O-[2-(methylamino)-2-oxoethyl] (2'-O-NMA). This modification resulted in an increase in the affinity of antisense oligonucleotides to complementary RNA similar to 2'-O-MOE-modified ASOs as compared to first-generation antisense oligodeoxynucleotides. The ASO modified with 2'-O-NMA reduced expression of PTEN mRNA in vitro and in vivo in a dose-dependent manner similar to 2'-O-MOE modified ASO. Importantly, toxicity parameters such as AST, ALT, organ weights, and body weights were found to be normal similar to 2'-O-MOE ASO-treated animal models. The data generated in these experiments suggest that 2'-O-NMA is a useful modification for potential application in both antisense and other oligonucleotide-based drug discovery efforts.

Publication types

  • Comparative Study

MeSH terms

  • Animals
  • Cell Line
  • Liver / cytology
  • Liver / metabolism
  • Male
  • Mice
  • Mice, Inbred BALB C
  • Nucleic Acid Hybridization
  • Oligoribonucleotides, Antisense / chemical synthesis*
  • Oligoribonucleotides, Antisense / chemistry
  • Oligoribonucleotides, Antisense / pharmacology
  • PTEN Phosphohydrolase / biosynthesis
  • PTEN Phosphohydrolase / genetics
  • RNA, Messenger / biosynthesis
  • Structure-Activity Relationship

Substances

  • Oligoribonucleotides, Antisense
  • RNA, Messenger
  • PTEN Phosphohydrolase