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Intravenous administration of scAAV9-Hexb normalizes lifespan and prevents pathology in Sandhoff disease mice.
Niemir, Natalia; Rouvière, Laura; Besse, Aurore; Vanier, Marie T; Dmytrus, Jasmin; Marais, Thibaut; Astord, Stéphanie; Puech, Jean-Philippe; Panasyuk, Ganna; Cooper, Jonathan D; Barkats, Martine; Caillaud, Catherine.
Afiliação
  • Niemir N; INSERM U1151, Institut Necker Enfants Malades, Paris 75014, France.
  • Rouvière L; Université Paris Descartes, Sorbonne Paris Cité, Paris, France.
  • Besse A; INSERM U1151, Institut Necker Enfants Malades, Paris 75014, France.
  • Vanier MT; Université Paris Descartes, Sorbonne Paris Cité, Paris, France.
  • Dmytrus J; Centre of Research in Myology, Institut de Myologie, Sorbonne Universités, UPMC University Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France.
  • Marais T; INSERM U820, Université de Lyon, Faculté de Médecine Lyon-Est, Lyon 69372, France.
  • Astord S; Department of Basic and Clinical Neuroscience, King's College London, Institute of Psychiatry, Psychology & Neuroscience, Maurice Wohl Clinical Neuroscience Institute, London SE5 9RX, UK.
  • Puech JP; Centre of Research in Myology, Institut de Myologie, Sorbonne Universités, UPMC University Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France.
  • Panasyuk G; Centre of Research in Myology, Institut de Myologie, Sorbonne Universités, UPMC University Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France.
  • Cooper JD; Service de Biochimie, Métabolomique et Protéomique, Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris 75015, France.
  • Barkats M; INSERM U1151, Institut Necker Enfants Malades, Paris 75014, France.
  • Caillaud C; Université Paris Descartes, Sorbonne Paris Cité, Paris, France.
Hum Mol Genet ; 27(6): 954-968, 2018 03 15.
Article em En | MEDLINE | ID: mdl-29325092
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Sandhoff / Hexosaminidase B Limite: Animals Idioma: En Ano de publicação: 2018 Tipo de documento: Article
Texto completo
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Sandhoff / Hexosaminidase B Limite: Animals Idioma: En Ano de publicação: 2018 Tipo de documento: Article