Chemical genetics is the investigation of proteins and signal transduction pathways by the screening of chemical libraries of small molecules. Chemical genetics is analogous to classical genetics where random mutations are introduced in organisms, the phenotype of these mutants is observed, and finally the specific gene mutation (genotype) that produced that phenotype is identified. In chemical genetics, the phenotype is disturbed not by introduction of mutations, but by exposure to small molecules (drug candidates). Phenotypic screening of chemical libraries is used to identify drug targets (forward chemical genetics) or to validate those targets in experimental models of disease (reverse chemical genetics). Recent applications of this topic have been implicated in signal transduction, which may play a role in discovering new cancer treatments.
"Chemical genetics", as the name implies, can serve as a unifying study between chemistry and biology. Pharmaceutical companies have recognized the potential here and are using high-throughput screening in combination with small molecule tools and chemistry to validate drug targets. In 2002, the National Cancer Institute founded the Initiative for Chemical Genetics (ICG), with the primary goal of discovering small molecule therapeutics. They aim to shrink the gap between discovery and therapeutic success and do so by making the information they discovered widely available. The ICG has published large amounts of data related to small molecules and chemical genetics publicly at ChemBank.
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