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Leveraging human genetics to guide drug target discovery

Nathan O Stitziel1, Sekar Kathiresan2

  • 1Cardiovascular Division, Department of Medicine, Washington University School of Medicine, Saint Louis, MO; Department of Genetics, Washington University School of Medicine, Saint Louis, MO; McDonnell Genome Institute, Washington University School of Medicine, Saint Louis, MO.

Trends in Cardiovascular Medicine
|October 1, 2016
PubMed

Insights

Human genetics studies accelerate drug discovery by identifying disease pathways and predicting drug efficacy and safety. This approach offers a more reliable method than traditional models for developing successful therapeutics.

Area of Science:

  • Pharmacology
  • Genetics
  • Epidemiology

Background:

  • Traditional drug development tools like observational epidemiology and animal models can be unreliable for identifying causal disease pathways.
  • Identifying effective molecular targets is crucial for successful therapeutic development.

Purpose of the Study:

  • To review the advantages of human genetics studies in accelerating drug target discovery.
  • To highlight how human genetics can improve the prediction of therapeutic efficacy and safety.

Main Methods:

  • Review of human genetics studies.
  • Analysis of genetic variation in relation to disease pathways and biomarkers.
  • Case examples from plasma lipid and coronary artery disease research.

Main Results:

  • Human genetics can identify disease-relevant pathways.
  • Genetic studies help discern causal links between biomarkers and disease.
  • Genetic variation predicts the efficacy and safety of molecular targets.

Conclusions:

  • Human genetics offers a powerful approach to accelerate drug discovery.
  • Leveraging human genetic data enhances the identification of reliable therapeutic targets.
  • This strategy can lead to more effective and safer drug development.
Keywords:
Cardiovascular diseaseHuman genetics

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