Overview of target validation and the impact of oligonucleotides

Simon W. Jones, Mark A. Lindsay

Research output: Contribution to journalReview articlepeer-review

8 Citations (SciVal)


During target validation, researchers attempt to modulate the activity of potential drug targets in relevant cell and/or animal disease models in order to identify those that might be expected to have therapeutic benefit in human patients. This has become increasingly important with the large expansion of potential targets identified by the human genome project and as a result of the spiralling costs of bringing drugs to market. This review will present an overview of the target-validation mechanism and examine the strengths and weaknesses of using oligonudeotide-based technologies such as antisense, short interfering RNA and aptamers.

Original languageEnglish
Pages (from-to)546-550
Number of pages5
JournalCurrent Opinion in Molecular Therapeutics
Issue number5
Publication statusPublished - Oct 2004


  • Antisense
  • Aptamers
  • Oligonucleotides
  • Short interfering RNA
  • Target validation

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics
  • Pharmacology
  • Drug Discovery
  • Genetics(clinical)


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