This technology is a self-regulating gene editing machinery system that limits the risk of off-target mutations and inflammatory responses, allowing for significantly safer gene therapy.
Gene editing machineries (GEMs) such as zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and RNA-guided engineered nucleases like CRISPR/Cas9 (RGENs) enable targeted manipulation of nucleic acid substrates via sequence recognition and enzymatic cleavage. A primary goal of each new generation of these GEMs has been to increase the target specificity. However, it is well accepted that prolonged nuclease activity can result in deleterious off-target mutations. Furthermore, inflammatory responses may be mounted against the nuclease proteins, and so a method is needed to limit the GEM activity after the target editing has taken place.
This technology is a self-cleaving GEM system that regulates the activity of an engineered nuclease. Expression of the cassette causes deactivation of the nuclease after completion of its desired editing activity, preventing sustained expression of the corresponding protein. This flexible technology can be tailored to regulate different types of GEM enzymes.
IR CU18335
Licensing Contact: Ron Katz