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Repeated AAV-mediated gene transfer by serotype switching enables long-lasting therapeutic levels of hUgt1a1 enzyme in a mouse model of Crigler-Najjar Syndrome Type I.
Bockor, L; Bortolussi, G; Iaconcig, A; Chiaruttini, G; Tiribelli, C; Giacca, M; Benvenuti, F; Zentilin, L; Muro, A F.
Afiliación
  • Bockor L; Mouse Molecular Genetics, Molecular Medicine and Cellular Immunology Groups, International Centre for Genetic Engineering and Biotechnology (ICGEB), Trieste, Italy.
  • Bortolussi G; Mouse Molecular Genetics, Molecular Medicine and Cellular Immunology Groups, International Centre for Genetic Engineering and Biotechnology (ICGEB), Trieste, Italy.
  • Iaconcig A; Mouse Molecular Genetics, Molecular Medicine and Cellular Immunology Groups, International Centre for Genetic Engineering and Biotechnology (ICGEB), Trieste, Italy.
  • Chiaruttini G; Mouse Molecular Genetics, Molecular Medicine and Cellular Immunology Groups, International Centre for Genetic Engineering and Biotechnology (ICGEB), Trieste, Italy.
  • Tiribelli C; Centro Studi Fegato, Fondazione Italiana Fegato, AREA Science Park Trieste, Italy.
  • Giacca M; Mouse Molecular Genetics, Molecular Medicine and Cellular Immunology Groups, International Centre for Genetic Engineering and Biotechnology (ICGEB), Trieste, Italy.
  • Benvenuti F; Mouse Molecular Genetics, Molecular Medicine and Cellular Immunology Groups, International Centre for Genetic Engineering and Biotechnology (ICGEB), Trieste, Italy.
  • Zentilin L; Mouse Molecular Genetics, Molecular Medicine and Cellular Immunology Groups, International Centre for Genetic Engineering and Biotechnology (ICGEB), Trieste, Italy.
  • Muro AF; Mouse Molecular Genetics, Molecular Medicine and Cellular Immunology Groups, International Centre for Genetic Engineering and Biotechnology (ICGEB), Trieste, Italy.
Gene Ther ; 24(10): 649-660, 2017 10.
Article en En | MEDLINE | ID: mdl-28805798
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Terapia Genética / Glucuronosiltransferasa / Dependovirus / Síndrome de Crigler-Najjar Tipo de estudio: Prognostic_studies Límite: Animals / Female / Humans / Male Idioma: En Año: 2017 Tipo del documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Terapia Genética / Glucuronosiltransferasa / Dependovirus / Síndrome de Crigler-Najjar Tipo de estudio: Prognostic_studies Límite: Animals / Female / Humans / Male Idioma: En Año: 2017 Tipo del documento: Article