The impact of CRISPR-Cas9 on target identification and validation

Drug Discov Today. 2015 Apr;20(4):450-7. doi: 10.1016/j.drudis.2014.12.016. Epub 2015 Jan 5.


The addition of an RNA-guided nuclease, Cas9, to the gene editing toolbox has increased the accessibility of gene editing technologies by greatly simplifying the design of editing reagents. Only a single 75-100 nucleotide RNA is required to guide Cas9 to the target gene of interest, which has meant that the established infrastructure of short-hairpin RNA interference screen could be readily adapted to genome-wide knock out screens. Cas9-based editing technology should streamline the generation of animal and cell-line models, make the generation of activity-dead mutations in target validation routine, and enable the discovery of a new generation of targets across therapeutic areas.

Publication types

  • Review

MeSH terms

  • Animals
  • Bacterial Proteins / genetics
  • Bacterial Proteins / metabolism*
  • CRISPR-Associated Protein 9
  • CRISPR-Cas Systems*
  • Clustered Regularly Interspaced Short Palindromic Repeats*
  • Drug Discovery / methods*
  • Endonucleases / genetics
  • Endonucleases / metabolism*
  • Gene Expression Regulation
  • Gene Targeting / methods*
  • Genetic Predisposition to Disease
  • Humans
  • Phenotype


  • Bacterial Proteins
  • CRISPR-Associated Protein 9
  • Cas9 protein, Francisella novicida
  • Endonucleases