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AAV-Mediated Gene Transfer Restores a Normal Muscle Transcriptome in a Canine Model of X-Linked Myotubular Myopathy.
Dupont, Jean-Baptiste; Guo, Jianjun; Renaud-Gabardos, Edith; Poulard, Karine; Latournerie, Virginie; Lawlor, Michael W; Grange, Robert W; Gray, John T; Buj-Bello, Ana; Childers, Martin K; Mack, David L.
Afiliação
  • Dupont JB; Department of Rehabilitation Medicine, Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA 98109, USA.
  • Guo J; Audentes Therapeutics, San Francisco, CA 94108, USA.
  • Renaud-Gabardos E; Genethon, INSERM UMR S951, Université Evry Val-d'Essone, Université Paris-Saclay, 91000 Evry, France.
  • Poulard K; Genethon, INSERM UMR S951, Université Evry Val-d'Essone, Université Paris-Saclay, 91000 Evry, France.
  • Latournerie V; Genethon, INSERM UMR S951, Université Evry Val-d'Essone, Université Paris-Saclay, 91000 Evry, France.
  • Lawlor MW; Department of Pathology and Laboratory Medicine and Neuroscience Research Center, Medical College of Wisconsin, Milwaukee, WI 53226, USA.
  • Grange RW; Department of Human Nutrition, Foods, and Exercise, Virginia Polytechnic Institute and State University, Blacksburg, VA 24060, USA.
  • Gray JT; Audentes Therapeutics, San Francisco, CA 94108, USA.
  • Buj-Bello A; Genethon, INSERM UMR S951, Université Evry Val-d'Essone, Université Paris-Saclay, 91000 Evry, France.
  • Childers MK; Department of Rehabilitation Medicine, Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA 98109, USA.
  • Mack DL; Department of Rehabilitation Medicine, Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA 98109, USA; Department of Bioengineering, University of Washington, Seattle, WA 98109, USA. Electronic address: dmack21@uw.edu.
Mol Ther ; 28(2): 382-393, 2020 02 05.
Article em En | MEDLINE | ID: mdl-31784415
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Técnicas de Transferência de Genes / Dependovirus / Músculo Esquelético / Miopatias Congênitas Estruturais / Transcriptoma / Vetores Genéticos Limite: Animals / Humans Idioma: En Revista: Mol Ther Ano de publicação: 2020 Tipo de documento: Article
Texto completo
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Técnicas de Transferência de Genes / Dependovirus / Músculo Esquelético / Miopatias Congênitas Estruturais / Transcriptoma / Vetores Genéticos Limite: Animals / Humans Idioma: En Revista: Mol Ther Ano de publicação: 2020 Tipo de documento: Article