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Target intelligence / Profile preview
ARCUS genome editor (ARCUS)
- Target
- ARCUS
- Molecular classification
- Engineered homing endonuclease, Genome editing enzyme
01
Overview
ARCUS is a proprietary genome editing technology developed by Precision BioSciences. It is an engineered genome editing platform based on the naturally occurring homing endonuclease I-CreI, originally found in the algae *Chlamydomonas reinhardtii*. ARCUS nucleases are designed to recognize specific DNA sequences and introduce double-stranded breaks, enabling precise gene insertion, correction, or replacement through homology-directed repair (HDR) or gene disruption/knockout through non-homologous end joining (NHEJ). Key features include its small size for efficient delivery, high sequence specificity to minimize off-target effects, and versatility to support various edit types.
02
Mechanism of action
Sequence-specific dsDNA cleavage with sticky ends, followed by HDR or NHEJ
03
Biological functions
Genome editingDNA cleavageGene insertionGene knockoutHomology-directed repair (HDR)Non-homologous end joining (NHEJ)
04
Disease associations
Chronic hepatitis B virus infectionPrimary mitochondrial myopathyGenetic disordersCancer
05
Safety considerations
Off-target effectsImmunogenicity
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