Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques.

Hordeaux, Juliette; Hinderer, Christian; Goode, Tamara; Buza, Elizabeth L; Bell, Peter; Calcedo, Roberto; Richman, Laura K; Wilson, James M

Hordeaux, Juliette; Hinderer, Christian; Goode, Tamara; Buza, Elizabeth L; Bell, Peter; Calcedo, Roberto; Richman, Laura K; Wilson, James M.

Afiliação

Hordeaux J; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA 19104, USA.
Hinderer C; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA 19104, USA.
Goode T; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA 19104, USA.
Buza EL; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA 19104, USA.
Bell P; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA 19104, USA.
Calcedo R; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA 19104, USA.
Richman LK; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA 19104, USA.
Wilson JM; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA 19104, USA.

Mol Ther Methods Clin Dev ; 10: 68-78, 2018 Sep 21.

Article em En | MEDLINE | ID: mdl-30073178

Palavras-chave

AAV9; MPS II; intrathecal

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Risk_factors_studies Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2018 Tipo de documento: Article

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