Template-assisted sequence knockin rescues skeletal and cardiac muscle function in a deletion model of Duchenne muscular dystrophy.

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Title: Template-assisted sequence knockin rescues skeletal and cardiac muscle function in a deletion model of Duchenne muscular dystrophy.
Authors: Fatehi S; Program in Genetics and Genome Biology, Peter Gilgan Centre for Research and Learning, Toronto, ON M5G 0A4, Canada; Department of Molecular Genetics, University of Toronto, Toronto, ON M5S 1A1, Canada., Rok MJ; Program in Genetics and Genome Biology, Peter Gilgan Centre for Research and Learning, Toronto, ON M5G 0A4, Canada; Department of Molecular Genetics, University of Toronto, Toronto, ON M5S 1A1, Canada., Marks RM; Program in Genetics and Genome Biology, Peter Gilgan Centre for Research and Learning, Toronto, ON M5G 0A4, Canada; Department of Molecular Genetics, University of Toronto, Toronto, ON M5S 1A1, Canada., Huynh E; Program in Genetics and Genome Biology, Peter Gilgan Centre for Research and Learning, Toronto, ON M5G 0A4, Canada., Kozman N; Program in Genetics and Genome Biology, Peter Gilgan Centre for Research and Learning, Toronto, ON M5G 0A4, Canada., Truong HA; Program in Genetics and Genome Biology, Peter Gilgan Centre for Research and Learning, Toronto, ON M5G 0A4, Canada., Chi L; Program in Translational Medicine, Peter Gilgan Centre for Research and Learning, Toronto, ON M5G 0A4, Canada., Yan B; Program in Translational Medicine, Peter Gilgan Centre for Research and Learning, Toronto, ON M5G 0A4, Canada., Khazeeva E; Program in Genetics and Genome Biology, Peter Gilgan Centre for Research and Learning, Toronto, ON M5G 0A4, Canada; Department of Molecular Genetics, University of Toronto, Toronto, ON M5S 1A1, Canada., Delgado-Olguin P; Program in Translational Medicine, Peter Gilgan Centre for Research and Learning, Toronto, ON M5G 0A4, Canada; Department of Molecular Genetics, University of Toronto, Toronto, ON M5S 1A1, Canada., Ivakine EA; Program in Genetics and Genome Biology, Peter Gilgan Centre for Research and Learning, Toronto, ON M5G 0A4, Canada; Department of Molecular Genetics, University of Toronto, Toronto, ON M5S 1A1, Canada; Department of Physiology, University of Toronto, Toronto, ON M5S 1A1, Canada. Electronic address: zhenya.ivakine@sickkids.ca., Cohn RD; Program in Genetics and Genome Biology, Peter Gilgan Centre for Research and Learning, Toronto, ON M5G 0A4, Canada; Department of Molecular Genetics, University of Toronto, Toronto, ON M5S 1A1, Canada; Department of Pediatrics, The Hospital for Sick Children, Toronto, ON M5G 1E8, Canada.
Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2025 Aug 06; Vol. 33 (8), pp. 3733-3745. Date of Electronic Publication: 2025 May 07.
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1525-0024
DOI:10.1016/j.ymthe.2025.05.005