CRISPR/Cas9 Editing to Facilitate and Expand Drug Discovery

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


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

Publication types

  • Review

MeSH terms

  • Animals
  • CRISPR-Cas Systems*
  • Drug Discovery / methods*
  • Drug Evaluation, Preclinical / methods
  • Drug Resistance / genetics
  • Gene Editing / methods*
  • Humans
  • Molecular Targeted Therapy / methods