Inhibition of telomerase by 2'-O-(2-methoxyethyl) RNA oligomers: effect of length, phosphorothioate substitution and time inside cells

Nucleic Acids Res. 2001 Apr 15;29(8):1683-9. doi: 10.1093/nar/29.8.1683.

Abstract

2'-O-(2-methoxyethyl) (2'-MOE) RNA possesses favorable pharmocokinetic properties that make it a promising option for the design of oligonucleotide drugs. Telomerase is a ribonucleoprotein that is up-regulated in many types of cancer, but its potential as a target for chemotherapy awaits the development of potent and selective inhibitors. Here we report inhibition of human telomerase by 2'-MOE RNA oligomers that are complementary to the RNA template region. Fully complementary oligomers inhibited telomerase in a cell extract with IC(50) values of 5-10 nM at 37 degrees C. IC(50) values for mismatch-containing oligomers varied with length and phosphorothioate substitution. After introduction into DU 145 prostate cancer cells inhibition of telomerase activity persisted for up to 7 days, equivalent to six population doublings. Inside cells discrimination between complementary and mismatch-containing oligomers increased over time. Our results reveal two oligomers as especially promising candidates for initiation of in vivo preclinical trials and emphasize that conclusions regarding oligonucleotide efficacy and specificity in cell extracts do not necessarily offer accurate predictions of activity inside cells.

Publication types

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

MeSH terms

  • Animals
  • Base Pair Mismatch / genetics
  • Base Pairing
  • Base Sequence
  • DNA, Recombinant / chemistry
  • DNA, Recombinant / genetics
  • DNA, Recombinant / metabolism
  • Enzyme Inhibitors / chemistry
  • Enzyme Inhibitors / metabolism*
  • Genetic Engineering
  • Humans
  • Inhibitory Concentration 50
  • Mice
  • Mice, Nude
  • Neoplasm Transplantation
  • Oligonucleotides / chemistry*
  • Oligonucleotides / genetics
  • Oligonucleotides / metabolism*
  • Oligoribonucleotides
  • RNA / chemistry
  • RNA / genetics
  • RNA / metabolism
  • RNA, Antisense / chemistry*
  • RNA, Antisense / genetics
  • RNA, Antisense / metabolism*
  • Substrate Specificity
  • Telomerase / antagonists & inhibitors*
  • Telomerase / genetics
  • Telomerase / metabolism
  • Time Factors
  • Transfection
  • Tumor Cells, Cultured

Substances

  • 2'-O-(2-methoxyethyl)-RNA
  • DNA, Recombinant
  • Enzyme Inhibitors
  • Oligonucleotides
  • Oligoribonucleotides
  • RNA, Antisense
  • RNA, recombinant
  • RNA
  • Telomerase