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AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington's Disease Minipig Model.
Evers, Melvin M; Miniarikova, Jana; Juhas, Stefan; Vallès, Astrid; Bohuslavova, Bozena; Juhasova, Jana; Skalnikova, Helena Kupcova; Vodicka, Petr; Valekova, Ivona; Brouwers, Cynthia; Blits, Bas; Lubelski, Jacek; Kovarova, Hana; Ellederova, Zdenka; van Deventer, Sander J; Petry, Harald; Motlik, Jan; Konstantinova, Pavlina.
Afiliación
  • Evers MM; Department of Research & Development, uniQure biopharma B.V., Amsterdam, the Netherlands. Electronic address: m.evers@uniqure.com.
  • Miniarikova J; Department of Research & Development, uniQure biopharma B.V., Amsterdam, the Netherlands.
  • Juhas S; Institute of Animal Physiology and Genetics, Libechov, Czech Republic.
  • Vallès A; Department of Research & Development, uniQure biopharma B.V., Amsterdam, the Netherlands.
  • Bohuslavova B; Institute of Animal Physiology and Genetics, Libechov, Czech Republic.
  • Juhasova J; Institute of Animal Physiology and Genetics, Libechov, Czech Republic.
  • Skalnikova HK; Institute of Animal Physiology and Genetics, Libechov, Czech Republic.
  • Vodicka P; Institute of Animal Physiology and Genetics, Libechov, Czech Republic.
  • Valekova I; Institute of Animal Physiology and Genetics, Libechov, Czech Republic.
  • Brouwers C; Department of Research & Development, uniQure biopharma B.V., Amsterdam, the Netherlands.
  • Blits B; Department of Research & Development, uniQure biopharma B.V., Amsterdam, the Netherlands.
  • Lubelski J; Department of Research & Development, uniQure biopharma B.V., Amsterdam, the Netherlands.
  • Kovarova H; Institute of Animal Physiology and Genetics, Libechov, Czech Republic.
  • Ellederova Z; Institute of Animal Physiology and Genetics, Libechov, Czech Republic.
  • van Deventer SJ; Department of Research & Development, uniQure biopharma B.V., Amsterdam, the Netherlands.
  • Petry H; Department of Research & Development, uniQure biopharma B.V., Amsterdam, the Netherlands.
  • Motlik J; Institute of Animal Physiology and Genetics, Libechov, Czech Republic.
  • Konstantinova P; Department of Research & Development, uniQure biopharma B.V., Amsterdam, the Netherlands.
Mol Ther ; 26(9): 2163-2177, 2018 09 05.
Article en En | MEDLINE | ID: mdl-30007561
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / Enfermedad de Huntington / Proteína Huntingtina Límite: Animals / Humans Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2018 Tipo del documento: Article Pais de publicación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / Enfermedad de Huntington / Proteína Huntingtina Límite: Animals / Humans Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2018 Tipo del documento: Article Pais de publicación: Estados Unidos