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click editor (CE2)
- Development stage
- Preclinical
- Lead developer
- Massachusetts General Hospital
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Intrathecal
01
Overview
Click editor (CE2) is a second-generation genome editing technology designed for precise genome writing. It consists of an RNA-programmable nickase (such as a CRISPR-Cas enzyme), a DNA-dependent DNA polymerase (DDP), and an HUH endonuclease (HUHe) domain. The HUHe domain facilitates the covalent attachment and recruitment of a single-stranded nucleic acid template (click DNA or clkDNA) that encodes the desired genetic modification. CE2 represents an optimization over the first-generation click editor (CE1) through protein engineering of the HUHe domain and chemical/structural optimization of the clkDNA substrate, resulting in significantly higher editing efficiency and potency at lower doses. The technology is being developed by researchers at Massachusetts General Hospital and the Technical University of Munich for potential therapeutic applications in genetic diseases.
- Other names
- second-generation click editor
02
Targets
Genomic DNA at the guide-RNA–specified locus
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