Directed evolution of compact RNA-guided nucleases for enhanced activity in mammalian cells

Abstract excerpt

RNA-guided nucleases enable DNA editing and offer promise for treating genetic diseases, particularly when used for precise sequence replacement. However, many of the most effective enzymes, such as S. pyogenes Cas9, are too large for delivery using vectors like adeno-associated virus (AAV). This has prompted interest in smaller alternatives from the Cas12f and TnpB families. Yet, these nucleases often show low a...

Topics

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• CRISPR and Genetic Engineering
• CRISPR-Associated Protein 9
• CRISPR-Associated Proteins
• CRISPR-Cas Systems
• Endonucleases
• Gene Editing
• HEK293 Cells
• Mutation

Identifiers and source

Literature Corpus work

c816365d-cf13-57cc-93fc-1a79be8dac1b

DOI

10.1101/2025.10.27.684765