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Correction of a splicing defect in a mouse model of congenital muscular dystrophy type 1A using a homology-directed-repair-independent mechanism.
Kemaladewi, Dwi U; Maino, Eleonora; Hyatt, Elzbieta; Hou, Huayun; Ding, Maylynn; Place, Kara M; Zhu, Xinyi; Bassi, Prabhpreet; Baghestani, Zahra; Deshwar, Amit G; Merico, Daniele; Xiong, Hui Y; Frey, Brendan J; Wilson, Michael D; Ivakine, Evgueni A; Cohn, Ronald D.
Affiliation
  • Kemaladewi DU; Program in Genetics and Genome Biology, The Hospital for Sick Children Research Institute, Toronto, Canada.
  • Maino E; Program in Genetics and Genome Biology, The Hospital for Sick Children Research Institute, Toronto, Canada.
  • Hyatt E; Department of Molecular Genetics, University of Toronto, Canada.
  • Hou H; Program in Genetics and Genome Biology, The Hospital for Sick Children Research Institute, Toronto, Canada.
  • Ding M; Program in Genetics and Genome Biology, The Hospital for Sick Children Research Institute, Toronto, Canada.
  • Place KM; Department of Molecular Genetics, University of Toronto, Canada.
  • Zhu X; Program in Genetics and Genome Biology, The Hospital for Sick Children Research Institute, Toronto, Canada.
  • Bassi P; Program in Genetics and Genome Biology, The Hospital for Sick Children Research Institute, Toronto, Canada.
  • Baghestani Z; Program in Genetics and Genome Biology, The Hospital for Sick Children Research Institute, Toronto, Canada.
  • Deshwar AG; Program in Genetics and Genome Biology, The Hospital for Sick Children Research Institute, Toronto, Canada.
  • Merico D; Department of Molecular Genetics, University of Toronto, Canada.
  • Xiong HY; Program in Genetics and Genome Biology, The Hospital for Sick Children Research Institute, Toronto, Canada.
  • Frey BJ; Deep Genomics, Toronto, Canada.
  • Wilson MD; Program in Genetics and Genome Biology, The Hospital for Sick Children Research Institute, Toronto, Canada.
  • Ivakine EA; Deep Genomics, Toronto, Canada.
  • Cohn RD; The Centre for Applied Genomics (TCAG), The Hospital for Sick Children Research Institute, Toronto, Canada.
Nat Med ; 23(8): 984-989, 2017 Aug.
Article in En | MEDLINE | ID: mdl-28714989
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: RNA, Messenger / Genetic Therapy / Laminin / RNA Splice Sites / DNA End-Joining Repair / Muscular Dystrophies Type of study: Risk_factors_studies Limits: Animals Language: En Journal: Nat Med Journal subject: BIOLOGIA MOLECULAR / MEDICINA Year: 2017 Document type: Article Affiliation country: Canada Country of publication: United States
Fulltext
Add to My VHL
Print
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PubMed Links
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: RNA, Messenger / Genetic Therapy / Laminin / RNA Splice Sites / DNA End-Joining Repair / Muscular Dystrophies Type of study: Risk_factors_studies Limits: Animals Language: En Journal: Nat Med Journal subject: BIOLOGIA MOLECULAR / MEDICINA Year: 2017 Document type: Article Affiliation country: Canada Country of publication: United States