Potent and selective inhibition of gene expression by an antisense heptanucleotide

Nat Biotechnol. 1996 Jul;14(7):840-4. doi: 10.1038/nbt0796-840.

Abstract

Factors that govern the specificity of an antisense oligonucleotide (ON) for its target RNA include accessibility of the targeted RNA to ON binding, stability of ON/RNA complexes in cells, and susceptibility of the ON/RNA complex to RNase H cleavage. ON specificity is generally proposed to be dependent on its length. To date, virtually all previous antisense experiments have used 12-25 nt-long ONs. We explored the antisense activity and specificity of short (7 and 8 nt) ONs modified with C-5 propyne pyrimidines and phosphorothioate internucleotide linkages. Gene-selective, mismatch sensitive, and RNase H-dependent inhibition was observed for a heptanucleotide ON. We demonstrated that the flanking sequences of the target RNA are a major determinant of specificity. The use of shorter ONs as antisense agents has the distinct advantage of simplified synthesis. These results may lead to a general, cost-effective solution to the development of antisense ONs as therapeutic agents.

MeSH terms

  • Animals
  • Base Sequence
  • Cell Line
  • Chlorocebus aethiops
  • Cost-Benefit Analysis
  • Gene Expression Regulation / drug effects*
  • HeLa Cells
  • Humans
  • Nucleic Acid Conformation
  • Oligonucleotides, Antisense / chemistry
  • Oligonucleotides, Antisense / metabolism
  • Oligonucleotides, Antisense / pharmacology*
  • RNA / chemistry
  • RNA / drug effects*
  • RNA / metabolism
  • Ribonuclease H / metabolism

Substances

  • Oligonucleotides, Antisense
  • RNA
  • Ribonuclease H