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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.
Afiliação
  • 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 em En | MEDLINE | ID: mdl-28714989

Texto completo: 1 Base de dados: MEDLINE Assunto principal: RNA Mensageiro / Terapia Genética / Laminina / Sítios de Splice de RNA / Reparo do DNA por Junção de Extremidades / Distrofias Musculares Idioma: En Ano de publicação: 2017 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: RNA Mensageiro / Terapia Genética / Laminina / Sítios de Splice de RNA / Reparo do DNA por Junção de Extremidades / Distrofias Musculares Idioma: En Ano de publicação: 2017 Tipo de documento: Article