Enhancement of template jump prime editing efficiency through cellular factor regulation
This research seeks to enhance the efficiency of template jump prime editing (TJ-PE) by identifying and regulating cellular factors involved in DNA repair. Using CRISPR screening, the study aims to improve the precision and effectiveness of TJ-PE for removing expanded repeats in patient cells.
Overview
The proposed solution focuses on advancing the efficiency of template jump prime editing (TJ-PE) by leveraging cellular factors involved in DNA repair processes. Currently, methods like Twin-PE, Prime-Del, and TJ-PE face challenges in efficiently removing extensive repeat lengths in patient cells. By conducting a comprehensive CRISPR screening to identify key DNA repair factors, the research aims to enhance TJ-PE's precision and effectiveness.
Technical specifications
Key features include:
- Use of pegRNA and nicking gRNA pairs for targeted editing
- CRISPRa and CRISPRi screening to identify DNA repair factors impacting TJ-PE
- Integration of a reporter cassette in engineered cell lines to monitor TJ-PE activity
- Use of next-generation sequencing to analyze gRNA changes post-TJ-PE
- Potential expansion to whole-genome screening to identify additional factors
Technology readiness level
The current stage of development is at TRL 2, indicating that the technology is in the early stages of experimental proof of concept, with ongoing efforts to identify and validate the impact of cellular factors on TJ-PE efficiency.
About GenKOre
GenKOre is a South Korean biotechnology company specializing in the development of gene editing therapies for the treatment of genetic diseases. The company is built upon its proprietary TaRGET (Tiny nuclease and augment RNA-based Genome Editing Technology) platform, which utilizes hypercompact CRISPR-Cas systems. This technology is designed to overcome the delivery limitations of traditional large-sized nucleases by enabling efficient packaging and delivery via adeno-associated virus (AAV) vectors. By combining high precision and delivery efficiency, the platform facilitates advanced genetic analysis, gene editing research, and the development of therapeutic solutions for human and animal health.
This technology is significant for addressing unmet medical needs in various genetic disorders, including neurological conditions like Huntington’s disease and ALS. GenKOre collaborates with global pharmaceutical companies and research institutions through joint research and technology transfer agreements to translate its platform into clinical applications. The company’s innovative R&D efforts, including the development of hypercompact base editing systems, have received recognition as national strategic technologies in South Korea, underscoring its role in advancing next-generation gene therapy. Through its specialized research centers, GenKOre continues to expand its therapeutic pipeline and strategic partnerships to bring precise, effective genetic medicines to market.
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