High-efficiency CRISPR-Cas nuclease
This technology is an optimized CRISPR-Cas12j nuclease for high-efficiency gene editing.
Unmet Need: Compact CRISPR-Cas nuclease for therapeutic applications
Current CRISPR-Cas nucleases face delivery challenges for therapeutic gene editing due to the trade-off between size and efficiency. Large, highly efficient nucleases cannot be packaged into single AAV vectors, which are required for targeted tissue delivery, while smaller AAV-compatible nucleases are generally less efficient. Thus, there is a clear need for CRISPR nucleases that are both compact and efficient for therapeutics.
The Technology: Optimized CRISPR-Cas nuclease for size and efficiency
This technology presents an optimized version of the CRISPR-Cas12j nuclease that enables more efficient gene editing. This Cas12j variant was engineered via a single amino acid mutation and has twice the efficiency compared to the wild-type nuclease. It maintains a size compatible with single AAV packaging, can be programmed to any target sequence with a nearby TTN, and has high fidelity.
Applications:
- Gene therapy for diseases requiring AAV delivery
- Ex vivo gene editing
- High throughput screening
- Drug discovery
- Research tool for modeling retinal disease
Advantages:
- 2x higher efficiency compared to wild-type Cas12j
- Compact size for single AAV packaging
- High fidelity with reduced off-targets
Lead Inventor:
Patent Information:
Patent Pending
Related Publications:
Tech Ventures Reference:
IR CU25094
Licensing Contact: Kristin Neuman