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PABPN1 gene therapy for oculopharyngeal muscular dystrophy.
Malerba, A; Klein, P; Bachtarzi, H; Jarmin, S A; Cordova, G; Ferry, A; Strings, V; Espinoza, M Polay; Mamchaoui, K; Blumen, S C; St Guily, J Lacau; Mouly, V; Graham, M; Butler-Browne, G; Suhy, D A; Trollet, C; Dickson, G.
Afiliação
  • Malerba A; School of Biological Sciences, Royal Holloway, University of London, Egham Hill, Egham, TW20 0EX Surrey, UK.
  • Klein P; Sorbonne Universités, UPMC Univ Paris 06, UM76, INSERM U974, Institut de Myologie, CNRS FRE3617, 47 bd de l'Hôpital, 75013 Paris, France.
  • Bachtarzi H; School of Biological Sciences, Royal Holloway, University of London, Egham Hill, Egham, TW20 0EX Surrey, UK.
  • Jarmin SA; School of Biological Sciences, Royal Holloway, University of London, Egham Hill, Egham, TW20 0EX Surrey, UK.
  • Cordova G; Sorbonne Universités, UPMC Univ Paris 06, UM76, INSERM U974, Institut de Myologie, CNRS FRE3617, 47 bd de l'Hôpital, 75013 Paris, France.
  • Ferry A; Sorbonne Universités, UPMC Univ Paris 06, UM76, INSERM U974, Institut de Myologie, CNRS FRE3617, 47 bd de l'Hôpital, 75013 Paris, France.
  • Strings V; Sorbonne Paris Cité, Université Paris Descartes, 75006 Paris, France.
  • Espinoza MP; Benitec Biopharma, 3940 Trust Way, Hayward, California 94545, USA.
  • Mamchaoui K; Sorbonne Universités, UPMC Univ Paris 06, UM76, INSERM U974, Institut de Myologie, CNRS FRE3617, 47 bd de l'Hôpital, 75013 Paris, France.
  • Blumen SC; Sorbonne Universités, UPMC Univ Paris 06, UM76, INSERM U974, Institut de Myologie, CNRS FRE3617, 47 bd de l'Hôpital, 75013 Paris, France.
  • St Guily JL; Department of Neurology, Hillel Yaffe Medical Center, Hadera and Rappaport Faculty of Medicine, The Technion, 1 Efron Street, Haifa 31096, Israel.
  • Mouly V; Sorbonne Universités, UPMC Univ Paris 06, UM76, INSERM U974, Institut de Myologie, CNRS FRE3617, 47 bd de l'Hôpital, 75013 Paris, France.
  • Graham M; Department of Otolaryngology-Head and Neck Surgery, Faculty of Medicine and University Pierre-et-Marie-Curie, Paris VI, Tenon Hospital, Assistance Publique des Hopitaux de Paris, 75252 Paris, France.
  • Butler-Browne G; Sorbonne Universités, UPMC Univ Paris 06, UM76, INSERM U974, Institut de Myologie, CNRS FRE3617, 47 bd de l'Hôpital, 75013 Paris, France.
  • Suhy DA; Benitec Biopharma, 3940 Trust Way, Hayward, California 94545, USA.
  • Trollet C; Sorbonne Universités, UPMC Univ Paris 06, UM76, INSERM U974, Institut de Myologie, CNRS FRE3617, 47 bd de l'Hôpital, 75013 Paris, France.
  • Dickson G; Benitec Biopharma, 3940 Trust Way, Hayward, California 94545, USA.
Nat Commun ; 8: 14848, 2017 03 31.
Article em En | MEDLINE | ID: mdl-28361972
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Mioblastos Esqueléticos / Distrofia Muscular Oculofaríngea / Proteína I de Ligação a Poli(A) / Força Muscular / Transcriptoma Limite: Animals / Humans Idioma: En Ano de publicação: 2017 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Mioblastos Esqueléticos / Distrofia Muscular Oculofaríngea / Proteína I de Ligação a Poli(A) / Força Muscular / Transcriptoma Limite: Animals / Humans Idioma: En Ano de publicação: 2017 Tipo de documento: Article