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Targeted genome editing in vivo corrects a Dmd duplication restoring wild-type dystrophin expression.
Maino, Eleonora; Wojtal, Daria; Evagelou, Sonia L; Farheen, Aiman; Wong, Tatianna W Y; Lindsay, Kyle; Scott, Ori; Rizvi, Samar Z; Hyatt, Elzbieta; Rok, Matthew; Visuvanathan, Shagana; Chiodo, Amanda; Schneeweiss, Michelle; Ivakine, Evgueni A; Cohn, Ronald D.
Affiliation
  • Maino E; Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, ON, Canada.
  • Wojtal D; Department of Molecular Genetics, University of Toronto, Toronto, ON, Canada.
  • Evagelou SL; Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, ON, Canada.
  • Farheen A; Department of Molecular Genetics, University of Toronto, Toronto, ON, Canada.
  • Wong TWY; Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, ON, Canada.
  • Lindsay K; Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, ON, Canada.
  • Scott O; Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, ON, Canada.
  • Rizvi SZ; Department of Molecular Genetics, University of Toronto, Toronto, ON, Canada.
  • Hyatt E; Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, ON, Canada.
  • Rok M; Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, ON, Canada.
  • Visuvanathan S; Institute of Medical Science, University of Toronto, Toronto, ON, Canada.
  • Chiodo A; Department of Pediatrics, the Hospital for Sick Children, Toronto, ON, Canada.
  • Schneeweiss M; Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, ON, Canada.
  • Ivakine EA; Department of Molecular Genetics, University of Toronto, Toronto, ON, Canada.
  • Cohn RD; Program in Genetics and Genome Biology, the Hospital for Sick Children Research Institute, Toronto, ON, Canada.
EMBO Mol Med ; 13(5): e13228, 2021 05 07.
Article in En | MEDLINE | ID: mdl-33724658
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Dystrophin / Muscular Dystrophy, Duchenne Type of study: Prognostic_studies Limits: Animals Language: En Journal: EMBO Mol Med Year: 2021 Document type: Article
Fulltext
Print
XML
PubMed Links
Search on Google

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Dystrophin / Muscular Dystrophy, Duchenne Type of study: Prognostic_studies Limits: Animals Language: En Journal: EMBO Mol Med Year: 2021 Document type: Article