This technology is an RNA templated genome editing technique that can be utilized in any organism, without the typical requirement for homologous recombination. Unmet Need: Genome editing without homologous recombination. Current CRISPR-based methods to edit the genome are dependent on homologous recombination, which may result in non-target deletions and insertions, translocations, and rearrangements.
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. “CRISPR genome surgery in a novel humanized model for autosomal dominant retinitis pigmentosa” Mol Ther. Unmet Need: Size-compatible genomic prime editing system.
