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Gene editing
(2025)Cite this article
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The ancestral protein of the CRISPR–Cas9 system — less than half the size of its famous descendant — is engineered into an effective DNA-targeting tool that can be delivered by AAV as an epigenome editor for in vivo gene silencing.
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Competing interests
C.A.G. is a co-founder of Tune Therapeutics and Locus Biosciences, and an advisor to Tune Therapeutics and Sarepta Therapeutics. C.A.G. and G.L.B. are inventors on patent applications related to genome and epigenome editing.
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Butterfield, G.L., Gersbach, C.A. Resurrecting a miniature Cas9 ancestor for genome and epigenome editing.
Nat Biotechnol (2025). https://doi.org/10.1038/s41587-025-02707-8
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DOI: https://doi.org/10.1038/s41587-025-02707-8
