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Curr Gene Ther. 2017;17(4):275-285. doi: 10.2174/1566523217666171121164615.

CRISPR/Cas9 Editing to Facilitate and Expand Drug Discovery.

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Department of Biochemistry, McGill University, Montreal, QC, H3G 1Y6, Canada.
Rosalind and Morris Goodman Cancer Research Center, McGill University, Montreal, QC, H3G 1Y6, Canada.
Department of Oncology, McGill University, Montreal, QC, H3G 1Y6, Canada.



The ability of most laboratories to easily access CRISPR/Cas9 engineering tools has caused a revolution in biology. One of the areas that will continue to be impacted by genome editing is the drug discovery process.


CRISPR/Cas9 will not only serve to accelerate the drug discovery pipeline, but also streamline line it by identifying high-value targets, facilitating the validation of drug: target interactions and mechanisms of action, and stimulating the development of phenotype-based high throughput screens as alternatives to target-based assays.


We review the literature and hurdles that have been overcome to develop the current generation of tools being used to enrich the drug discovery paradigm.


Base editing; CRISPR/Cas9; Drug discovery; Druggable genome; RNA; Target: drug interaction validation

[Indexed for MEDLINE]

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