Cornerstones of CRISPR-Cas in drug discovery and therapy

Nat Rev Drug Discov. 2017 Feb;16(2):89-100. doi: 10.1038/nrd.2016.238. Epub 2016 Dec 23.


The recent development of CRISPR-Cas systems as easily accessible and programmable tools for genome editing and regulation is spurring a revolution in biology. Paired with the rapid expansion of reference and personalized genomic sequence information, technologies based on CRISPR-Cas are enabling nearly unlimited genetic manipulation, even in previously difficult contexts, including human cells. Although much attention has focused on the potential of CRISPR-Cas to cure Mendelian diseases, the technology also holds promise to transform the development of therapies to treat complex heritable and somatic disorders. In this Review, we discuss how CRISPR-Cas can affect the next generation of drugs by accelerating the identification and validation of high-value targets, uncovering high-confidence biomarkers and developing differentiated breakthrough therapies. We focus on the promises, pitfalls and hurdles of this revolutionary gene-editing technology, discuss key aspects of different CRISPR-Cas screening platforms and offer our perspectives on the best practices in genome engineering.

Publication types

  • Review

MeSH terms

  • Animals
  • Biomarkers
  • CRISPR-Associated Proteins / drug effects*
  • CRISPR-Cas Systems
  • Clustered Regularly Interspaced Short Palindromic Repeats / drug effects*
  • Drug Discovery / trends*
  • Drug Therapy / trends*
  • Genetic Engineering
  • Genetic Therapy / trends
  • Humans


  • Biomarkers
  • CRISPR-Associated Proteins