Precision BioSciences publishes ARCUS nucleases data

Precision BioSciences (DTIL) announced the publication of a peer-reviewed manuscript titled “High-Efficiency Homology-Directed Insertion into the Genome using the Engineered Homing Endonuclease ARCUS” in the journal Nucleic Acids Research. This publication demonstrates a wide variety of gene edits including high-frequency transgene insertions using ARCUS nucleases to stimulate a homology dependent repair mechanism. The publication provides evidence to understand the repair mechanism and how ARCUS can produce high insertion frequencies in both dividing and non-dividing cells. Highlights from the publication include: ARCUS nucleases support rates of transgene insertion exceeding 85% in T lymphocytes via homology-directed repair. Through its unique ability to generate 3′ overhang ends at the DNA break, ARCUS can facilitate transgene insertion in up to 40% of non-dividing primary human hepatocytes. ARCUS editing by HDR can also be used to achieve other gene editing objectives such as gene insertion, single base editing, specific small and large deletions, and replacement of large stretches of genomic DNA which could enable editing of genes that are too large for gene therapy approaches. Mechanistic studies demonstrate the necessity of the 3′ overhang that ARCUS nucleases create to drive homology-mediated gene insertion in dividing and non-dividing cells.