Nature biotechnology
CRISPR-Cas3-based editing for targeted deletions in a mouse model of transthyretin amyloidosis.
Saeko Ishida, Yusuke Sato, Keisuke Chosa, Eri Ezawa, Yuko Yamauchi, Masaaki Oyama, Hiroko Kozuka-Hata, Rina Ito, Rikako Sato, Masatoshi Maeki, Tomo-O Ishikawa, Kenichi Yamamura, Kohei Takeshita, Kensuke Yamaguchi, Yuta Kochi
Published: 202610.1038/s41587-025-02949-6
Abstract
CRISPR-Cas3 represents a mechanistically distinct genome-editing system compared to Cas9 that generates long-range deletions rather than small indels, thereby reducing the risk of residual protein function from in-frame mutations. Here we evaluated C…
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