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Cas9-induced single cut enables highly efficient and template-free repair of a muscular dystrophy causing founder mutation.

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第一作者： Stefanie,Müthel

第一单位： Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association (MDC), 13125 Berlin, Germany.;Muscle Research Unit at the Experimental and Clinical Research Center, a Cooperation Between the Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association (MDC) and the Charité-Universitätsmedizin Berlin, 13125 Berlin, Germany.;Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Experimental and Clinical Research Center, 10117 Berlin, Germany.

作者： Stefanie,Müthel ^[1] ; Andreas,Marg ^[1] ; Busem,Ignak ^[1] ; Janine,Kieshauer ^[1] ; Helena,Escobar ^[1] ; Christian,Stadelmann ^[2] ; Simone,Spuler ^[2]

作者单位： Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association (MDC), 13125 Berlin, Germany.;Muscle Research Unit at the Experimental and Clinical Research Center, a Cooperation Between the Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association (MDC) and the Charité-Universitätsmedizin Berlin, 13125 Berlin, Germany.;Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Experimental and Clinical Research Center, 10117 Berlin, Germany. ^[1] Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association (MDC), 13125 Berlin, Germany.;Muscle Research Unit at the Experimental and Clinical Research Center, a Cooperation Between the Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association (MDC) and the Charité-Universitätsmedizin Berlin, 13125 Berlin, Germany.;Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Experimental and Clinical Research Center, 10117 Berlin, Germany.;Department of Biology, Chemistry and Pharmacy, Freie Universität Berlin, 14195 Berlin, Germany. ^[2]

关键词 CRISPR-Cas9 LGMD MT: RNA/DNA Editing gene editing mRNA stem cell therapy

DOI 10.1016/j.omtn.2023.02.005

PMID 36865086

发布时间 2024-09-13

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