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.

To learn more about our gene editing work, read the USA Today article, ‘Gene Editing Institute pioneering DNA surgery.’

For more information on the Gene Editing Institute and Wistar Institute partnership, read ‘Graham Cancer Center and Wistar Institute to expand gene editing research.’

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.

For more information, call 302-623-6028 or e-mail

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.

Our Current Research Program

We have undertaken the decidedly reductionist approach for studying the mechanism of action and regulation of gene editing. The Gene Editing Institute continues to evaluate the overall role of DNA damage response proteins in the process of genome modification with a particular emphasis on how double strand breaks and the phases of the cell cycle influence the efficiency and efficacy of gene editing in mammalian cells, directed by single-stranded oligonucleotides. We are also analyzing how specific chemical modification of nucleotides that are placed within the single-stranded DNA oligonucleotide can improve the frequency with which gene editing takes place. We are using single-stranded DNA oligonucleotides and CRISPR/Cas9 systems to recapitulate the chromosomal translocations in childhood leukemias in HEK 293 cells, a model system that affords the opportunity to evaluate changes in global gene expression and epigenetic modification as a function of chromosomal exchange.