Application

BioTherapeutics

BioTools
PlantSciences

DNE is the "missing piece" between functional genomics research and clinical medicine. Engineered meganucleases produced by Precision BioSciences are being developed to enable the precise in situ correction of genes responsible for human disease. The use of DNE as an in vivo or ex vivo gene therapy avoids many of the pitfalls of existing methods, for example using DNE:

• Avoids Transgene Silencing. Gene correction using DNE ensures stable and heritable gene expression that is resistant to gene silencing over time.
• Insures Appropriate Gene Expression. Gene correction using DNE ensures that therapeutic genes are expressed from their endogenous promoters, which ensures appropriate expression levels, mRNA splicing variation, and tissue specificity.
• Prevents Insertional Mutagenesis. Gene correction using DNE avoids insertional mutagenesis due to random integration of a therapeutic transgene. It is this property of conventional gene therapy strategies, and its frequently tragic outcome, that must be addressed before genetic therapy finds a place in clinical medicine.