The virtuous cycle of human genetics and mouse models in drug discovery

Joseph H Nadeau1, Johan Auwerx2

  • 1Pacific Northwest Research Institute, Seattle, WA, USA. jnadeau@pnri.org.

Insights

Mouse models are crucial for understanding disease mechanisms and developing treatments, despite some skepticism. Integrating human studies with rigorous animal research accelerates therapeutic discovery.

Area of Science:

  • Genetics and Developmental Biology
  • Pharmacology and Therapeutics
  • Comparative Medicine

Background:

  • Phenotypic variation is studied across species to find disease intervention targets.
  • Concerns exist regarding the reliability of drug targets identified in mouse models for human application.

Purpose of the Study:

  • To review evidence supporting the continued importance of mouse models in biomedical research.
  • To advocate for integrating human observational studies with animal model experiments.

Main Methods:

  • Review of existing scientific literature and evidence.
  • Analysis of contributions from mouse models to understanding genetic principles and disease.

Main Results:

  • Mouse models have made fundamental contributions to genetics, pathogenic mechanisms, and therapeutic strategies.
  • Evidence supports the value of mouse models in biomedical research.

Conclusions:

  • Mouse models remain essential for advancing our understanding of biology and disease.
  • An integrated approach combining human studies and model organism research fosters a cycle of discovery.

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