Application of antisense oligonucleotides for gene functionalization and target validation

Curr Opin Mol Ther. 1999 Jun;1(3):359-71.


The Human Genome Project (complete sequencing of the human genome) will be complete soon and the information made available to the biomedical community. Although the project is not yet complete, it has dramatically changed the practice of biomedical sciences. With enormous amounts of information available from sequencing efforts, increasing demands are being put on researchers to quickly determine the biochemical function of novel molecular targets and to validate them as appropriate for drug discovery endeavors. Antisense oligonucleotides are an ideal technology for gene functionalization and target validation. They are an efficient methodology for gene functionalization and target validation and are a proven technology. Antisense technology can answer questions with a high degree of precision and it is a versatile technology. In this review the use of antisense oligonucleotides as a research tool for gene functionalization and target validation is discussed.

Publication types

  • Review

MeSH terms

  • Animals
  • Drug Delivery Systems
  • Drug Design
  • Drug Evaluation / methods*
  • Drug Industry / methods
  • Gene Expression / drug effects
  • Genes / physiology
  • Human Genome Project
  • Humans
  • Isoenzymes / antagonists & inhibitors
  • Isoenzymes / genetics
  • Kinetics
  • Oligonucleotides, Antisense / chemical synthesis
  • Oligonucleotides, Antisense / chemistry
  • Oligonucleotides, Antisense / pharmacokinetics
  • Oligonucleotides, Antisense / pharmacology*
  • Oncogenes
  • Protein Kinase C / antagonists & inhibitors
  • Protein Kinase C / genetics
  • RNA, Messenger / antagonists & inhibitors
  • RNA, Messenger / chemistry
  • RNA, Messenger / genetics
  • Signal Transduction / drug effects
  • Structure-Activity Relationship


  • Isoenzymes
  • Oligonucleotides, Antisense
  • RNA, Messenger
  • Protein Kinase C