Generation and utility of genetically humanized mouse models

Drug Discov Today. 2013 Dec;18(23-24):1200-11. doi: 10.1016/j.drudis.2013.07.007. Epub 2013 Jul 18.


Identifying in vivo models that are naturally predictive for particular areas of study in humans can be challenging due to the divergence that has occurred during speciation. One solution to this challenge that is gaining increasing traction is the use of genetic engineering to introduce human genes into mice to generate superior models for predicting human responses. This review describes the state-of-the-art for generating such models, provides an overview of the types of genetically humanized mouse models described to date and their applications in basic research, drug discovery and development and to understand clinical drug toxicity. We discuss limitations and explore promising future directions for the use of genetically humanized mice to further improve translational research.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Animals, Genetically Modified
  • Drug Design*
  • Drug Discovery / methods
  • Drug-Related Side Effects and Adverse Reactions*
  • Genetic Engineering / methods*
  • Humans
  • Mice
  • Mice, Transgenic
  • Models, Animal
  • Translational Research, Biomedical / methods