"CRISPR–Cas3-based editing for targeted deletions in a mouse model of transthyretin amyloidosis"
Nature Biotechnology Online 05 January 2026 doi:10.1038/s41587-025-02949-6
New Study Explores Therapeutic Potential of CRISPR-Cas3 Genome-Editing System
Description
Published Article
Saeko Ishida1, Yusuke Sato2, Keisuke Chosa1,3, Eri Ezawa1, Yuko Yamauchi1, Masaaki Oyama4, Hiroko Kozuka-Hata4, Rina Ito2, Rikako Sato2, Masatoshi Maeki5, Tomo-o Ishikawa6, Kenichi Yamamura7, Kohei Takeshita8,9, Kensuke Yamaguchi10, Yuta Kochi11, Fumitaka Hashiya12, Yiwei Liu13, Naoko Abe13, Hiroshi Abe13,14, Yoshiki Sekijima15, Kazuto Yoshimi1,16, and Tomoji Mashimo1,16
1Division of Animal Genetics, Laboratory Animal Research Center, Institute of Medical Science, The University of Tokyo, Japan
2Laboratory for Molecular Design of Pharmaceutics, Faculty of Pharmaceutical Sciences, Hokkaido University, Japan
3C4U Corporation, Japan
4Medical Proteomics Laboratory, Institute of Medical Science, The University of Tokyo, Japan
5Division of Applied Chemistry, Faculty of Engineering, Hokkaido University, Japan
6Transgenic, Inc., Japan
7Transgenic Group, Inc., Japan
8Life Science Research Infrastructure Group, Advanced Photon Technology Division, RIKEN SPring-8 Center, Japan
9Department of Life Science, University of Hyogo, Japan
10Biomedical Engineering Research Innovation Center, Laboratory for Biomaterials and Bioengineering, Institute of Integrated Research, Institute of Science Tokyo, Japan
11Department of Genomic Function and Diversity, Medical Research Laboratory, Institute of Integrated Research, Institute of Science Tokyo, Japan
12Research Center for Materials Science, Nagoya University, Japan
13Department of Chemistry, Graduate School of Science, Nagoya University, Japan
14Institute for Glyco-core Research (iGCORE), Nagoya University, Japan
15Department of Medicine (Neurology and Rheumatology), Shinshu University School of Medicine, Japan
16Division of Genome Engineering, Center for Experimental Medicine and Systems Biology, Institute of Medical Science, The University of Tokyo, Japan
1Division of Animal Genetics, Laboratory Animal Research Center, Institute of Medical Science, The University of Tokyo, Japan
2Laboratory for Molecular Design of Pharmaceutics, Faculty of Pharmaceutical Sciences, Hokkaido University, Japan
3C4U Corporation, Japan
4Medical Proteomics Laboratory, Institute of Medical Science, The University of Tokyo, Japan
5Division of Applied Chemistry, Faculty of Engineering, Hokkaido University, Japan
6Transgenic, Inc., Japan
7Transgenic Group, Inc., Japan
8Life Science Research Infrastructure Group, Advanced Photon Technology Division, RIKEN SPring-8 Center, Japan
9Department of Life Science, University of Hyogo, Japan
10Biomedical Engineering Research Innovation Center, Laboratory for Biomaterials and Bioengineering, Institute of Integrated Research, Institute of Science Tokyo, Japan
11Department of Genomic Function and Diversity, Medical Research Laboratory, Institute of Integrated Research, Institute of Science Tokyo, Japan
12Research Center for Materials Science, Nagoya University, Japan
13Department of Chemistry, Graduate School of Science, Nagoya University, Japan
14Institute for Glyco-core Research (iGCORE), Nagoya University, Japan
15Department of Medicine (Neurology and Rheumatology), Shinshu University School of Medicine, Japan
16Division of Genome Engineering, Center for Experimental Medicine and Systems Biology, Institute of Medical Science, The University of Tokyo, Japan
