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A Novel Small NPC1 Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann-Pick Type C1 Disease.
Hughes, Michael Paul; Nelvagal, Hemanth Ramesh; Coombe-Tennant, Oliver; Smith, Dave; Smith, Claire; Massaro, Giulia; Poupon-Bejuit, Laura; Platt, Frances Mary; Rahim, Ahad Abdul.
Afiliação
  • Hughes MP; UCL School of Pharmacy, University College London, London WC1N 1AX, UK.
  • Nelvagal HR; UCL School of Pharmacy, University College London, London WC1N 1AX, UK.
  • Coombe-Tennant O; UCL School of Pharmacy, University College London, London WC1N 1AX, UK.
  • Smith D; Department of Pharmacology, University of Oxford, Oxford OX1 3QT, UK.
  • Smith C; Department of Pharmacology, University of Oxford, Oxford OX1 3QT, UK.
  • Massaro G; UCL School of Pharmacy, University College London, London WC1N 1AX, UK.
  • Poupon-Bejuit L; UCL School of Pharmacy, University College London, London WC1N 1AX, UK.
  • Platt FM; Department of Pharmacology, University of Oxford, Oxford OX1 3QT, UK.
  • Rahim AA; UCL School of Pharmacy, University College London, London WC1N 1AX, UK.
Cells ; 12(12)2023 06 13.
Article em En | MEDLINE | ID: mdl-37371089
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Peptídeos e Proteínas de Sinalização Intracelular / Doença de Niemann-Pick Tipo C / Proteína C1 de Niemann-Pick Limite: Animals Idioma: En Ano de publicação: 2023 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Peptídeos e Proteínas de Sinalização Intracelular / Doença de Niemann-Pick Tipo C / Proteína C1 de Niemann-Pick Limite: Animals Idioma: En Ano de publicação: 2023 Tipo de documento: Article