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Established PABPN1 intranuclear inclusions in OPMD muscle can be efficiently reversed by AAV-mediated knockdown and replacement of mutant expanded PABPN1.
Malerba, Alberto; Klein, Pierre; Lu-Nguyen, Ngoc; Cappellari, Ornella; Strings-Ufombah, Vanessa; Harbaran, Sonal; Roelvink, Peter; Suhy, David; Trollet, Capucine; Dickson, George.
Afiliação
  • Malerba A; Centres of Gene and Cell Therapy and Biomedical Sciences, School of Biological Sciences, Royal Holloway, University of London, Egham TW20 0EX, Surrey, UK.
  • Klein P; Sorbonne Université, INSERM, Association Institut de Myologie, Centre de Recherche en Myologie, UMRS974, 47 bd de l'Hôpital, 75013 Paris, France.
  • Lu-Nguyen N; Centres of Gene and Cell Therapy and Biomedical Sciences, School of Biological Sciences, Royal Holloway, University of London, Egham TW20 0EX, Surrey, UK.
  • Cappellari O; Comparative Biomedical Sciences, Royal Veterinary College, London NW1 0TU, UK.
  • Strings-Ufombah V; Benitec Biopharma, Hayward, CA 94545, USA.
  • Harbaran S; Benitec Biopharma, Hayward, CA 94545, USA.
  • Roelvink P; Benitec Biopharma, Hayward, CA 94545, USA.
  • Suhy D; Benitec Biopharma, Hayward, CA 94545, USA.
  • Trollet C; Sorbonne Université, INSERM, Association Institut de Myologie, Centre de Recherche en Myologie, UMRS974, 47 bd de l'Hôpital, 75013 Paris, France.
  • Dickson G; Centres of Gene and Cell Therapy and Biomedical Sciences, School of Biological Sciences, Royal Holloway, University of London, Egham TW20 0EX, Surrey, UK.
Hum Mol Genet ; 28(19): 3301-3308, 2019 10 01.
Article em En | MEDLINE | ID: mdl-31294444
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Dependovirus / Distrofia Muscular Oculofaríngea / Proteína I de Ligação a Poli(A) / Corpos de Inclusão Intranuclear Limite: Animals / Humans Idioma: En Revista: Hum Mol Genet Assunto da revista: BIOLOGIA MOLECULAR / GENETICA MEDICA Ano de publicação: 2019 Tipo de documento: Article País de afiliação: Reino Unido
Texto completo
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XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Dependovirus / Distrofia Muscular Oculofaríngea / Proteína I de Ligação a Poli(A) / Corpos de Inclusão Intranuclear Limite: Animals / Humans Idioma: En Revista: Hum Mol Genet Assunto da revista: BIOLOGIA MOLECULAR / GENETICA MEDICA Ano de publicação: 2019 Tipo de documento: Article País de afiliação: Reino Unido