Crystal structure and improved antisense properties of 2'-O-(2-methoxyethyl)-RNA

Nat Struct Biol. 1999 Jun;6(6):535-9. doi: 10.1038/9304.

Abstract

2'-O-(2-Methoxyethyl)-RNA (MOE-RNA) is a nucleic acid analog with promising features for antisense applications. Compared with phosphorothioate DNA (PS-DNA), the MOE modification offers improved nuclease resistance, enhanced RNA affinity, improved cellular uptake and intestinal absorption, reduced toxicity and immune stimulation. The crystal structure of a fully modified MOE-RNA dodecamer duplex (CGCGAAUUCGCG) was determined at 1.7 A resolution. In the majority of the MOE substituents, the torsion angle around the ethylene alkyl chain assumes a gauche conformation. The conformational preorganization of the MOE groups is consistent with the improved RNA affinity and the extensive hydration of the substituents could play a role in the improved cellular uptake of MOE-RNA. A specific hydration pattern that bridges substituent and phosphate oxygen atoms in the minor groove of MOE-RNA may explain its high nuclease resistance.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Base Pairing
  • Base Sequence
  • Crystallization
  • Crystallography, X-Ray
  • Drug Design
  • Ethylenes / chemistry
  • Ethylenes / metabolism
  • Hydrogen Bonding
  • Models, Molecular
  • Nucleic Acid Conformation*
  • Oligoribonucleotides
  • Oxygen / metabolism
  • Phosphates / metabolism
  • RNA, Antisense / chemistry*
  • RNA, Antisense / genetics
  • RNA, Antisense / metabolism
  • RNA, Antisense / therapeutic use
  • Structure-Activity Relationship
  • Water / metabolism

Substances

  • 2'-O-(2-methoxyethyl)-RNA
  • Ethylenes
  • Oligoribonucleotides
  • Phosphates
  • RNA, Antisense
  • Water
  • ethylene
  • Oxygen