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Nature Research, Nature Biotechnology, 4(30), p. 317-320, 2012

DOI: 10.1038/nbt.2151

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Use of genome-wide association studies for drug repositioning

This paper is available in a repository.
This paper is available in a repository.

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Abstract

Over the past few years, large investments have been made in genome-wide association studies (GWAS) with the expectation that some of these studies would lead to the identification of novel therapeutic modalities or allow selection of patients who would respond better to therapeutic interventions. Although the results have provided valuable biological insights for many common diseases, the translation of the genetics findings from GWAS into the clinic remains limited and a topic of intense debate. Among the factors that could explain this situation are that the road from a gene target to an approved marketed drug takes in general more than ten years and most GWAS results have only been obtained over the past four years. Furthermore, because the effect size of the common variants identified by GWAS, alone or in aggregation, is generally modest, the impact in terms of personalized, individually tailored medicine has been negligible. We present here an analysis of another potential application of GWAS data—drug repositioning. In the following study, we assess the utility of GWAS in systematically and rapidly identifying alternative or refined indications for existing drugs.