Using model-system genetics for drug-based target discovery

The combination of medicinal chemistry and model-organism genetics is emerg ing as a powerful tool for the discovery and validation of drug targets. Mo del systems can be used to identify the cognate target for compounds that d emonstrate in vivo efficacy but have unknown mechanisms of action. Alternat ively, drugs with known cognate targets can be used to probe biochemical pa thways in model organisms, revealing new targets and mechanisms within thes e pathways. In both cases, the availability of human genomic sequence data is opening up new opportunities for accelerating target discovery.