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Iterative engineering of a compact Cas9 ortholog for <i>in vivo</i> gene editing via single AAV delivery

2026-01-12

Abstract excerpt

<h4>ABSTRACT</h4> The development of compact and efficient CRISPR-Cas systems is crucial for biomedical and therapeutic genome editing, particularly in vivo applications based on viral delivery. Here, we performed a comparative functional screen of seven Cas9 orthologs to systematically evaluate their genome editing activity in mammalian cells. Among these, Cme2—a 1008 amino acid nuclease recognizing a 5′-NAGNGC...

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Literature Corpus work
7a691664-a555-57f2-813b-ad9e12a5c581
DOI
10.64898/2026.01.12.698990
Open publication

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Iterative engineering of a compact Cas9 ortholog for <i>in vivo</i> gene editing via single AAV deliveryDOI 10.64898/2026.01.12.698990
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