The recent development of CRISPR technology for targeted genomic editing may enable disruptive advances in biology and medicine in a very short time frame. Derived from viral-mediated defense systems, Cas9-mediated genomic engineering together with rapidly developing synthetic biology tools are applicable to model systems, plants, animals, and even human cells.

It is now possible to introduce mutations to evaluate gene function, generate models of human genetic disease, perform gene correction, chromosomally integrate synthetic gene networks that perform complex regulatory functions, and redesign genome structure or build whole genomes from scratch. In the near term, gene editing could revolutionize the way preclinical research is carried out, enabling custom-designed safety models (e.g. humanized rats), highly engineered cell lines to meld target and phenotypic screening, and synthetic systems for enhanced drug production. In the longer term, precise replacement of one sequence with another holds promise as a therapeutic application in diseases such as muscular dystrophy and severe combined immunodeficiency. Dr. Korn will discuss how CRISPR-Cas9 was discovered, how it was turned into a gene editing technology, recent results uncovering mechanisms by which cells repair Cas9-induced DNA breaks, and will introduce a clinical project in hematological disease.