Leveraging human genetics to guide drug target discovery

Nathan O. Stitziel, Sekar Kathiresan

Research output: Contribution to journalReview article

18 Scopus citations

Abstract

Identifying appropriate molecular targets is a critical step in drug development. Despite many advantages, the traditional tools of observational epidemiology and cellular or animal models of disease can be misleading in identifying causal pathways likely to lead to successful therapeutics. Here, we review some favorable aspects of human genetics studies that have the potential to accelerate drug target discovery. These include using genetic studies to identify pathways relevant to human disease, leveraging human genetics to discern causal relationships between biomarkers and disease, and studying genetic variation in humans to predict the potential efficacy and safety of inhibitory compounds aimed at molecular targets. We present some examples taken from studies of plasma lipids and coronary artery disease to highlight how human genetics can accelerate therapeutics development.

Original languageEnglish
Pages (from-to)352-359
Number of pages8
JournalTrends in Cardiovascular Medicine
Volume27
Issue number5
DOIs
StatePublished - Jul 2017

Keywords

  • Cardiovascular disease
  • Human genetics

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