579. Meganucleases as an Efficient Tool for Genome Editing

The field of genome editing has exploded in recent years. However, despite being the only nuclease naturally evolved for genome editing, meganucleases have largely remained behind the scenes compared to ZFNs, TALENs, and CRISPRs due to difficulties in modifying their DNA-recognition specificity. We have developed a next-generation meganuclease platform called “ARCUS” that overcomes these production difficulties and can produce nucleases with customized activity and specificity. Unlike some other genome editing technologies, ARCUS nucleases are capable of distinguishing target sites that only differ by 1 base pair. We will present the ARCUS platform and one such example of a meganuclease engineered to distinguish the dominant P23H point mutation in the rhodopsin gene from its wild type allele for a possible gene therapy of retinitis pigmentosa