Gene Editing Institute Advancing Gene Editing and Genome Engineering for More Than 20 Years
The mission of the Gene Editing Institute is to elucidate the mechanism and regulation of genome editing at the molecular level and to help translate our results into clinically relevant applications. We employ single-stranded oligonucleotides and programmable nucleases, including TALENs and CRISPRS, as genetic tools to direct nucleotide exchange at specific sites within the mammalian genome. We use primary and model cell systems to study how gene editing takes place and to identify the activators and suppressors that control the reaction.
Translation of our work will lead to the development of effective therapeutics for the treatment of inherited diseases as well as for the creation of credible and robust cell lines that can be used for the screening of novel anticancer drugs.
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20 Years of Discovery in Gene Editing
The Gene Editing Institute is widely recognized as a pioneering laboratory in the field of gene editing both in lower and higher eukaryotes. Over the course of the last 20 years, the Institute has focused on understanding the basic mechanism and regulation of gene editing within the context of the chromosome. These investigations, detailed in the above timeline, included analysis at the biochemical, molecular and cellular levels and generated a series of discoveries that led to the eventual development of specialized oligonucleotides (chimeras) that could direct targeted gene correction. These observations led to the conceptualization of the process of gene editing in mammalian cells.