Simple Genetics for a Complex Disease

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Science  10 May 2013:
Vol. 340, Issue 6133, pp. 689-690
DOI: 10.1126/science.1239101

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The cost of bringing a drug to market is staggering (estimated at more than 1 billion dollars) and the failure rate is daunting: Only one in three drugs that reach phase 3 clinical trials ultimately reach the marketplace. Accordingly, there has been considerable interest in improving strategies to predict the effects of new therapeutic agents. The outcome of several recent trials of a new class of cholesterol-lowering agents—antibodies against an enzyme called proprotein convertase subtilisin kexin type 9 (PCSK9) (1) — illustrates the potential role of human genetics not only in identifying drug targets, but also in predicting the likely consequences of specific interventions.