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Target discovery

Abstract

Target discovery, which involves the identification and early validation of disease-modifying targets, is an essential first step in the drug discovery pipeline. Indeed, the drive to determine protein function has been stimulated, both in industry and academia, by the completion of the human genome project. In this article, we critically examine the strategies and methodologies used for both the identification and validation of disease-relevant proteins. In particular, we will examine the likely impact of recent technological advances, including genomics, proteomics, small interfering RNA and mouse knockout models, and conclude by speculating on future trends.

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Figure 1: Overview of molecular- and system-based approaches to target discovery.
Figure 2: Correlative technologies used in target identification.
Figure 3: Phenotype-driven target identification.
Figure 4: Overview of the techniques used in target validation.

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Online Mendelian Inheritance in Man

cystic fibrosis

Duchenne muscular dystrophy

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Lindsay, M. Target discovery. Nat Rev Drug Discov 2, 831–838 (2003). https://doi.org/10.1038/nrd1202

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