Related Experiment Video
Updated: Oct 9, 2026

A Manual Small Molecule Screen Approaching High-throughput Using Zebrafish Embryos
Published on: November 8, 2014
Using model-system genetics for drug-based target discovery
1Exelixis, 170 Harbor Way, South San 94083 0511, Francisco, CA, USA
Abstract:
The combination of medicinal chemistry and model-organism genetics is emerging as a powerful tool for the discovery and validation of drug targets. Model systems can be used to identify the cognate target for compounds that demonstrate in vivo efficacy but have unknown mechanisms of action. Alternatively, drugs with known cognate targets can be used to probe biochemical pathways in model organisms, revealing new targets and mechanisms within these pathways. In both cases, the availability of human genomic sequence data is opening up new opportunities for accelerating target discovery.
Related Concept Videos
Pharmacogenomics: Identification of New Drug Targets
Drug Discovery: Overview
Genetic Screens
Forward genetic screens
Forward or “classical” genetic screens involve creating random mutations in an organism’s DNA using radiation, mutagens, or insertion of additional bases, which result in visible changes...
Pharmacogenetic Phenotypes: Alterations in Pharmacokinetics, Drug Targets and Biologic Milieu
Pharmacodynamic Models: Emax Drug–Concentration Effect Model
Pharmacodynamic Models: Overview
