title: The impact of CRISPR-Cas9 on target identification and validation.
abstract: 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.
authors.affiliation: Horizon Discovery, 7100 Cambridge Research Park, Waterbeach, Cambridge CB25 9TL, UK. Electronic address: j.moore@horizondiscovery.com.
authors.forename: Jonathan D
authors.initials: JD
authors.lastname: Moore
mesh_terms: D000818:Animals; D001426:Bacterial Proteins / Q000235:genetics / Q000378:metabolism*; D000076987:CRISPR-Associated Protein 9; D064113:CRISPR-Cas Systems; D064112:Clustered Regularly Interspaced Short Palindromic Repeats; D055808:Drug Discovery / Q000379:methods*; D004720:Endonucleases / Q000235:genetics / Q000378:metabolism*; D005786:Gene Expression Regulation; D018390:Gene Targeting / Q000379:methods*; D020022:Genetic Predisposition to Disease; D006801:Humans; D010641:Phenotype
chemical_list: D001426:Bacterial Proteins; D000076987:CRISPR-Associated Protein 9; C000600049:Cas9 protein, Francisella novicida; D004720:Endonucleases