Abstract

Introduction: 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.

Objective: 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.

Conclusion: 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.

Keywords: CRISPR/Cas9, Drug discovery, Target: drug interaction validation, Base editing, Druggable genome, RNA.