Article

Single-AAV CRISPR editing of skeletal muscle in non-human primates with NanoCas, an ultracompact nuclease

2025-01-30

Abstract excerpt

CRISPR gene editing is a transformative technology for addressing genetic diseases, but delivery constraints have largely limited its therapeutic applications to liver-targeted and ex vivo therapies. Here, we present the discovery and engineering of NanoCas, an ultracompact CRISPR nuclease capable of extending CRISPR’s reach in vivo beyond liver targets. We experimentally screened 176 ultracompact CRISPR systems...

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Literature Corpus work: 37a7a07b-3498-5186-93cc-a4dd09b2bca5
DOI: 10.1101/2025.01.29.635576

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