Discussion

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 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 is streamlining the generation of animal and cell-line models, making the generation of activity-dead mutations in target validation routine, and promises to enable the discovery of a new generation of targets across therapeutic areas. My talk will focus on how Horizon is deploying CRISPR to perform knock-out and overexpression screens to find new targets for cancer therapies, understand resistance mechanisms and will outline how this technology can be used in other therapeutic areas.