Summary

Description

Results from the Human Genome Project revealed that the human genome contains 20,000 to 25,000 genes. A gene contains (encodes) the information that each cell uses to make (express) a protein, which is essential for the body to function properly. Abnormal protein expression is associated with many human diseases, which makes proteins key targets for therapeutic agents. Approximately 3,000 genes are considered part of the "druggable genome," a set of genes encoding proteins that scientists can or predict they can modulate using experimental small molecule compounds. Yet only about 10 percent of these genes encode proteins that have been targeted successfully by an approved drug. Therefore, a large number of proteins remain for scientists to explore as potential therapeutic targets. The vast majority of the druggable genome encodes four key protein families: G-protein-coupled receptors, nuclear receptors, ion channels and kinases. Researchers lack crucial knowledge about the function of many proteins from these families and their roles in health and disease. Better understanding of how these proteins work could shed light on new avenues of investigation for basic science and therapeutic discovery.