Antisense oligonucleotide therapy in cancer

Curr Opin Mol Ther. 2003 Apr;5(2):118-22.


The sequencing of the human genome has highlighted some of the genes that are of importance in disease states. This has provided opportunities for the development of new therapeutics to target a wide range of human diseases. These new drugs are intended to be highly specific; antisense oligonucleotides (ONs) are one such class of new drugs. ONs are short pieces of DNA which hybridize to a specific target mRNA blocking its translation to protein, thereby inhibiting the action of the gene. Several genes known to be of importance in the regulation of apoptosis, cell growth, metastasis and angiogenesis provide a tantalizing prospect for the development of anticancer agents. The phosphorothioate antisense ONs are the current choice for antisense therapy. This article reviews the current strategies for antisense targets in cancer therapy.

Publication types

  • Review

MeSH terms

  • Apoptosis / drug effects
  • Clusterin
  • Genes, bcl-2 / drug effects
  • Genes, bcl-2 / genetics*
  • Glycoproteins / drug effects
  • Humans
  • Molecular Chaperones / drug effects
  • Neoplasms / drug therapy*
  • Neoplasms / genetics
  • Oligonucleotides, Antisense / therapeutic use*
  • Protein Kinase C / drug effects


  • CLU protein, human
  • Clusterin
  • Glycoproteins
  • Molecular Chaperones
  • Oligonucleotides, Antisense
  • Protein Kinase C