Genetic-Driven Druggable Target Identification and Validation

Trends Genet. 2018 Jul;34(7):558-570. doi: 10.1016/j.tig.2018.04.004. Epub 2018 May 23.

Abstract

Choosing the right biological target is the critical primary decision for the development of new drugs. Systematic genetic association testing of both human diseases and quantitative traits, along with resultant findings of coincident associations between them, is becoming a powerful approach to infer drug targetable candidates and generate in vitro tests to identify compounds that can modulate them therapeutically. Here, we discuss opportunities and challenges, and infer criteria for the optimal use of genetic findings in the drug discovery pipeline.

Keywords: coincident genetic associations; drug discovery; druggability; quantitative phenotypes; therapeutic targets.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, N.I.H., Intramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Drug Discovery / methods*
  • Humans
  • Quantitative Trait Loci / genetics*