Mouse model phenotypes provide information about human drug targets

Bioinformatics. 2014 Mar 1;30(5):719-25. doi: 10.1093/bioinformatics/btt613. Epub 2013 Oct 24.


Motivation: Methods for computational drug target identification use information from diverse information sources to predict or prioritize drug targets for known drugs. One set of resources that has been relatively neglected for drug repurposing is animal model phenotype.

Results: We investigate the use of mouse model phenotypes for drug target identification. To achieve this goal, we first integrate mouse model phenotypes and drug effects, and then systematically compare the phenotypic similarity between mouse models and drug effect profiles. We find a high similarity between phenotypes resulting from loss-of-function mutations and drug effects resulting from the inhibition of a protein through a drug action, and demonstrate how this approach can be used to suggest candidate drug targets.

Availability and implementation: Analysis code and supplementary data files are available on the project Web site at

MeSH terms

  • Animals
  • Cyclooxygenase 2 Inhibitors / pharmacology
  • Diclofenac / pharmacology
  • Drug Repositioning / methods*
  • Humans
  • Mice
  • Mice, Knockout
  • Models, Animal
  • Phenotype*
  • Proteins / antagonists & inhibitors*
  • Proteins / classification
  • Proteins / drug effects


  • Cyclooxygenase 2 Inhibitors
  • Proteins
  • Diclofenac