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AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington's disease.
Miniarikova, J; Zimmer, V; Martier, R; Brouwers, C C; Pythoud, C; Richetin, K; Rey, M; Lubelski, J; Evers, M M; van Deventer, S J; Petry, H; Déglon, N; Konstantinova, P.
Afiliación
  • Miniarikova J; Department of Research & Development, uniQure N.V., Amsterdam, The Netherlands.
  • Zimmer V; Department of Gastroenterology and Hepatology, Leiden University Medical Center, Leiden, The Netherlands.
  • Martier R; Neurosciences Research Center (CRN), Laboratory of Cellular and Molecular Neurotherapies (LCMN), Lausanne University Hospital, Lausanne, Switzerland.
  • Brouwers CC; Department of Clinical Neurosciences, LCMN, Lausanne University Hospital, Lausanne, Switzerland.
  • Pythoud C; Department of Research & Development, uniQure N.V., Amsterdam, The Netherlands.
  • Richetin K; Department of Gastroenterology and Hepatology, Leiden University Medical Center, Leiden, The Netherlands.
  • Rey M; Department of Research & Development, uniQure N.V., Amsterdam, The Netherlands.
  • Lubelski J; Neurosciences Research Center (CRN), Laboratory of Cellular and Molecular Neurotherapies (LCMN), Lausanne University Hospital, Lausanne, Switzerland.
  • Evers MM; Department of Clinical Neurosciences, LCMN, Lausanne University Hospital, Lausanne, Switzerland.
  • van Deventer SJ; Neurosciences Research Center (CRN), Laboratory of Cellular and Molecular Neurotherapies (LCMN), Lausanne University Hospital, Lausanne, Switzerland.
  • Petry H; Department of Clinical Neurosciences, LCMN, Lausanne University Hospital, Lausanne, Switzerland.
  • Déglon N; Neurosciences Research Center (CRN), Laboratory of Cellular and Molecular Neurotherapies (LCMN), Lausanne University Hospital, Lausanne, Switzerland.
  • Konstantinova P; Department of Clinical Neurosciences, LCMN, Lausanne University Hospital, Lausanne, Switzerland.
Gene Ther ; 24(10): 630-639, 2017 10.
Article en En | MEDLINE | ID: mdl-28771234

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Proteínas Nucleares / Enfermedad de Huntington / Tratamiento con ARN de Interferencia / Proteínas del Tejido Nervioso Límite: Animals / Humans / Male Idioma: En Revista: Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2017 Tipo del documento: Article País de afiliación: Países Bajos

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Proteínas Nucleares / Enfermedad de Huntington / Tratamiento con ARN de Interferencia / Proteínas del Tejido Nervioso Límite: Animals / Humans / Male Idioma: En Revista: Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2017 Tipo del documento: Article País de afiliación: Países Bajos