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Closing the Protein Knowledge Gap to Identify New Treatments

Screenshot of the NIH Pharos website.

Screenshot of the NIH Pharos website.

Each of the 20,000 genes that make up the human genome carries a set of instructions that a cell can use to make a protein. These proteins play many critical roles in human health and disease: They can transmit signals between organs, transport other molecules throughout the body and protect cells from an invading virus. But as many as 3,000 genes are considered part of the “druggable genome,” a set of genes encoding proteins that scientists already can or predict they can modify by using experimental compounds.

As part of the NIH Common Fund’s Illuminating the Druggable Genome (IDG) program, a team of NCATS and other scientists recently discovered that about one in every three proteins is poorly understood. To speed this research along, NCATS scientists developed Pharos, an online portal that provides access to the protein information collected by IDG investigators. This resource is designed to help address the lack of preliminary data available for understudied proteins, in turn helping scientists further their research to close the protein knowledge gap. Read more in Nature Reviews Drug Discovery.

“Our results are a starting point for future research to gain a better understanding of these understudied proteins, which can lead to new or improved treatment approaches,” said study co-author Tudor Oprea, M.D., Ph.D., of the University of New Mexico.

Posted April 2018