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Updated: Aug 5, 2025

Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
Published on: December 11, 2016
Future prospects for human genetics and genomics in drug discovery
Maya Ghoussaini1, Matthew R Nelson2, Ian Dunham3
1Wellcome Sanger Institute, Wellcome Genome Campus, United Kingdom; Open Targets, Wellcome Genome Campus, United Kingdom. Electronic address: https://twitter.com/MayaGhoussaini.
Abstract:
Evidence from human genetics supporting the therapeutic hypothesis increases the likelihood that a drug will succeed in clinical trials. Rare and common disease genetics yield a wide array of alleles with a range of effect sizes that can proxy for the effect of a drug in disease. Recent advances in large scale population collections and whole genome sequencing approaches have provided a rich resource of human genetic evidence to support drug target selection. As the range of phenotypes profiled increases and ever more alleles are discovered across world-wide populations, these approaches will increasingly influence multiple stages across the lifespan of a drug discovery programme.
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