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Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy.
Amoasii, Leonela; Hildyard, John C W; Li, Hui; Sanchez-Ortiz, Efrain; Mireault, Alex; Caballero, Daniel; Harron, Rachel; Stathopoulou, Thaleia-Rengina; Massey, Claire; Shelton, John M; Bassel-Duby, Rhonda; Piercy, Richard J; Olson, Eric N.
Afiliação
  • Amoasii L; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Hildyard JCW; Exonics Therapeutics, 75 Kneeland Street, Boston, MA 02111, USA.
  • Li H; Department of Clinical Science and Services, Comparative Neuromuscular Diseases Laboratory, Royal Veterinary College, London NW1 0TU, UK.
  • Sanchez-Ortiz E; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Mireault A; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Caballero D; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Harron R; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Stathopoulou TR; Department of Clinical Science and Services, Comparative Neuromuscular Diseases Laboratory, Royal Veterinary College, London NW1 0TU, UK.
  • Massey C; Section of Anaesthesia and Analgesia, Royal Veterinary College, London NW1 0TU, UK.
  • Shelton JM; Department of Clinical Science and Services, Comparative Neuromuscular Diseases Laboratory, Royal Veterinary College, London NW1 0TU, UK.
  • Bassel-Duby R; Department of Internal Medicine, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Piercy RJ; Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.
  • Olson EN; Department of Clinical Science and Services, Comparative Neuromuscular Diseases Laboratory, Royal Veterinary College, London NW1 0TU, UK.
Science ; 362(6410): 86-91, 2018 10 05.
Article em En | MEDLINE | ID: mdl-30166439

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Distrofina / Distrofia Muscular de Duchenne / Edição de Genes Limite: Animals Idioma: En Ano de publicação: 2018 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Distrofina / Distrofia Muscular de Duchenne / Edição de Genes Limite: Animals Idioma: En Ano de publicação: 2018 Tipo de documento: Article