Chemical genetics: ligand-based discovery of gene function

Nat Rev Genet. 2000 Nov;1(2):116-25. doi: 10.1038/35038557.


Chemical genetics is the study of gene-product function in a cellular or organismal context using exogenous ligands. In this approach, small molecules that bind directly to proteins are used to alter protein function, enabling a kinetic analysis of the in vivo consequences of these changes. Recent advances have strongly enhanced the power of exogenous ligands such that they can resemble genetic mutations in terms of their general applicability and target specificity. The growing sophistication of this approach raises the possibility of its application to any biological process.

Publication types

  • Review

MeSH terms

  • Animals
  • Databases as Topic
  • Gene Library
  • Genes*
  • Ligands
  • Molecular Biology / methods*
  • Peptide Library*
  • Proteins / genetics*


  • Ligands
  • Peptide Library
  • Proteins