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Chem Commun (Camb). 2017 Jun 29;53(53):7162-7167. doi: 10.1039/c7cc02349a.

Elucidating drug targets and mechanisms of action by genetic screens in mammalian cells.

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Institute for Neurodegenerative Diseases, Department of Biochemistry and Biophysics, University of California, San Francisco and Chan Zuckerberg Biohub, San Francisco, California, USA.


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.

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