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AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease.
Hughes, Michael P; Smith, Dave A; Morris, Lauren; Fletcher, Claire; Colaco, Alexandria; Huebecker, Mylene; Tordo, Julie; Palomar, Nuria; Massaro, Giulia; Henckaerts, Els; Waddington, Simon N; Platt, Frances M; Rahim, Ahad A.
Afiliación
  • Hughes MP; Department of Pharmacology, UCL School of Pharmacy, University College London, London WC1N 1AX, UK.
  • Smith DA; Department of Pharmacology, University of Oxford, Oxford OX13QT, UK.
  • Morris L; Department of Pharmacology, University of Oxford, Oxford OX13QT, UK.
  • Fletcher C; Department of Pharmacology, University of Oxford, Oxford OX13QT, UK.
  • Colaco A; Department of Pharmacology, University of Oxford, Oxford OX13QT, UK.
  • Huebecker M; Department of Pharmacology, University of Oxford, Oxford OX13QT, UK.
  • Tordo J; Department of Infectious Diseases, School of Immunology and Microbial Sciences, King's College London, London SE19RT, UK.
  • Palomar N; Department of Infectious Diseases, School of Immunology and Microbial Sciences, King's College London, London SE19RT, UK.
  • Massaro G; Department of Pharmacology, UCL School of Pharmacy, University College London, London WC1N 1AX, UK.
  • Henckaerts E; Department of Infectious Diseases, School of Immunology and Microbial Sciences, King's College London, London SE19RT, UK.
  • Waddington SN; Gene Transfer Technology Group, UCL Institute for Women's Health, University College London, London WC1E 6HX, UK.
  • Platt FM; Department of Pharmacology, University of Oxford, Oxford OX13QT, UK.
  • Rahim AA; Department of Pharmacology, UCL School of Pharmacy, University College London, London WC1N 1AX, UK.
Hum Mol Genet ; 27(17): 3079-3098, 2018 09 01.
Article en En | MEDLINE | ID: mdl-29878115
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Glicoproteínas de Membrana / Terapia Genética / Proteínas Portadoras / Adenoviridae / Trastornos Neurológicos de la Marcha / Modelos Animales de Enfermedad / Enfermedad de Niemann-Pick Tipo C / Longevidad Tipo de estudio: Prognostic_studies Límite: Animals / Humans Idioma: En Revista: Hum Mol Genet Asunto de la revista: BIOLOGIA MOLECULAR / GENETICA MEDICA Año: 2018 Tipo del documento: Article País de afiliación: Reino Unido
Texto completo
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Glicoproteínas de Membrana / Terapia Genética / Proteínas Portadoras / Adenoviridae / Trastornos Neurológicos de la Marcha / Modelos Animales de Enfermedad / Enfermedad de Niemann-Pick Tipo C / Longevidad Tipo de estudio: Prognostic_studies Límite: Animals / Humans Idioma: En Revista: Hum Mol Genet Asunto de la revista: BIOLOGIA MOLECULAR / GENETICA MEDICA Año: 2018 Tipo del documento: Article País de afiliación: Reino Unido