This technology is a DNA editing platform with a reduced payload size to allow adeno-associated virus (AAV) and nanoparticle packaging for gene therapy.
Mutations in short sequences or single bases in the human genome account for 89% of known pathogenic variants, implying a need for efficient and directable genomic editing. Current methods to edit small regions of DNA through a process called “prime editing” are contained within a DNA payload that is too large to facilitate many gene therapies. Reducing the size of the prime editing platform would permit packaging into a single adeno-associated virus (AAV), which would allow for safer and more efficient delivery strategies and expand the application of gene therapy to treat a broader variety of conditions.
This technology enables enhanced modification of a target nucleic acid by using a single subunit of the reverse transcriptase. By presenting a smaller size than conventional prime editors, this technology can be packed and delivered in a single adeno-associated virus (AAV). The smaller payload allows for more effective delivery of genetic therapeutics and could also be used in other genetic assays, such as RT-PCR. Delivery of the technology enhances the ability of researchers and clinicians to design, optimize, and test novel gene therapies for adaptation into next-generation therapeutics.
This technology has been validated with in vitro studies with different cell lines.
Patent Pending (US20250171810)
IR CU22084
Licensing Contact: Kristin Neuman
