2. Venue : B101 , Institute of Biomedical Science and Technology
3. Speaker : Hyongbum Kim, MD, PhD
Genome editing using programmable nucleases
Genetic modification of cells is important to enhance the therapeutic efficacy. Programmable nucleases, which include zinc-finger nucleases (ZFNs), transcriptional activator-like effector nucleases (TALENs), and RNA-guided engineered nucleases (RGENs) derived from the prokaryotic CRISPR–Cas system, enable targeted genetic modifications in cultured cells, animals and plants. The great value of these enzymes in cell therapy arises from their ability to induce site-specific DNA cleavages in the genome, the repair of which (via endogenous mechanisms) enables high-precision genome editing. However, these nucleases differ from each other in several respects, including composition, targetable sites, specificities and mutation signatures, among other characteristics. Knowledge of nuclease-specific features as well as their pros and cons is essential for researchers to choose the most appropriate tool for genetic modification of cells for transplantation.