How we produce a precise genetic alteration

A custom DNE nuclease can be produced and validated in vitro within two weeks.  Depending on the application, the DNE nuclease or the gene encoding it can be introduced into a cell using readily available delivery methods.  The nuclease then cleaves its intended site in the genomic DNA to stimulate gene modification as part of the DNA repair process.  Eukaryotic cells repair double-strand DNA breaks through two different processes: 1) non-homologous end-joining (NHEJ) and 2) homologous recombination.  By taking advantage of these natural processes, DNE technology allows us to target virtually any genetic change within a large genome.

For in vivo applications such as transgenics and gene therapy, meganuclease exposure is transient but results in a permanent, heritable chromosomal modification.  This allows us to produce a desired phenotype without eliciting an immune response or integrating unwanted genetic material into the genome.