Target identification and validation through genetics

Abstract

Drug target selection and validation is the goal of commercial genetic research. Human studies are beginning to deliver on their early promise of identifying complex disease susceptibility genes as the first step in validating potential new targets. Although the genes identified to date have shed light on some of the biological mechanisms that underlie complex disease, they have not always been tractable as drug targets. The current genetic strategies for disease gene identification are now being applied to specifically identify genetically associated drug targets in a high-throughput manner.