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Genome Editing in Drug Discovery


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       Klio Maratou1, Aaron T. Cheng2, Fiona M. Behan1, Ning Sun2, and Quinn Lu3

       1 Functional Genomics, R&D GlaxoSmithKline, Stevenage, UK

       2 Functional Genomics, R&D GlaxoSmithKline, Upper Providence, PA, USA

       3 Novel Human Genetics Research Unit, R&D GlaxoSmithKline, Upper Providence, PA, USA

      CRISPR genome editing technologies provide versatile tools for the genetic manipulation and screening of genes and pathways in mammalian cells and in model animals. Their applications in drug discovery are broad, including target discovery, target validation, mechanism of action, and target engagement studies (Lu et al. 2017). Since first described, many improvements and novel applications of the technology have been reported. The technologies include gene knock out (KO) via non‐homologous end joining (NHEJ) following CRISPR‐mediated double‐stranded break (DSB), gene knock in (KI) for SNP/mutation generation