TitleElucidating drug targets and mechanisms of action by genetic screens in mammalian cells.
Publication TypeJournal Article
Year of Publication2017
AuthorsKampmann M
JournalChem Commun (Camb)
Date Published2017 Jun 29
KeywordsAnimals, Cell Survival, Clustered Regularly Interspaced Short Palindromic Repeats, Genetic Testing, Humans, Neoplasms, Phenotype, Small Molecule Libraries

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.

Alternate JournalChem. Commun. (Camb.)
PubMed ID28487920
PubMed Central IDPMC5507204
Grant ListDP2 GM119139 / GM / NIGMS NIH HHS / United States
K99 CA181494 / CA / NCI NIH HHS / United States
R00 CA181494 / CA / NCI NIH HHS / United States