8 Chemical genetics

Chemical genetics is based on the ability of small molecules to bind reversibly to biological molecules resulting in a phenotypic change in cells. Recent advances in the synthesis of combinatorial libraries of diverse skeletal structure along with the use of cell-based high throughput screening has led to the identification of novel molecules highly targeted towards specific pathways. Screening of pharmaceutical libraries has revealed novel and unexpected combinations of molecules effective against cancer and other diseases. The number of possible molecules less than 3000 daltons is truly vast and it is with chemical genetics that “chemical space” can be fully explored.