Triage therapeutic candidates
Our models are generated using a highly optimized parental donor which has been proven to display extraordinary differentiation characteristics which yield robust, reliable and reproducible hepatocytes after gene editing.
The result is a set of unique disease models with the same genetic background, but CRISPR gene edited to introduce a pathogenic mutation for the disease being investigated.
DefiniGEN’s scientific team are adept at analyzing a wide range of therapeutic modalities which have been proven to work reproducibly in these cell models including small molecules, AAV, lentiviral vectors, siRNA, antisense oligonucleotides, mRNA, plasmid and a variety of base editing therapeutic approaches.
