Elucidating drug targets and mechanisms of action by genetic screens in mammalian cells. Issue 53 (10th May 2017)

• Record Type: Journal Article
• Authors: Kampmann, Martin
• Is Part Of: Chemical communications. Volume 53:Issue 53(2017)
• Page Start: 7162
• Page End: 7167
• Publication Date: 2017-05-10
• Subjects: Chemistry -- Periodicals; 540
• DOI: 10.1039/c7cc02349a
• Languages: English
• ISSNs: 1359-7345

Abstract

Abstract : Genome-wide CRISPR interference (CRISPRi) screens in mammalian cells enable drug target identification and uncover genes controlling drug response. Abstract : Phenotypic screening is a powerful approach to discover small molecules with desired effects on biological systems, which can then be developed into therapeutic drugs. The identification of the target and mechanism of action of compounds discovered in phenotypic screens remains a major challenge. This feature article describes the use of genetic tools to reveal drug targets and mechanisms in mammalian cells. Until recently, RNA interference was the method of choice for such studies. Here, we highlight very recent additions to the genetic toolkit in mammalian cells, including CRISPR, CRISPR interference, and CRISPR activation, and illustrate their usefulness for drug target identification.