Dual-function AAV gene therapy reverses late-stage Canavan disease pathology in mice.

Fröhlich, Dominik; Kalotay, Elizabeth; von Jonquieres, Georg; Bongers, Andre; Lee, Brendan; Suchowerska, Alexandra K; Housley, Gary D; Klugmann, Matthias

Fröhlich, Dominik; Kalotay, Elizabeth; von Jonquieres, Georg; Bongers, Andre; Lee, Brendan; Suchowerska, Alexandra K; Housley, Gary D; Klugmann, Matthias.

Afiliación

Fröhlich D; Translational Neuroscience Facility and Department of Physiology, School of Biomedical Sciences, University of New South Wales, Sydney, NSW, Australia.
Kalotay E; Translational Neuroscience Facility and Department of Physiology, School of Biomedical Sciences, University of New South Wales, Sydney, NSW, Australia.
von Jonquieres G; Translational Neuroscience Facility and Department of Physiology, School of Biomedical Sciences, University of New South Wales, Sydney, NSW, Australia.
Bongers A; Biological Resources Imaging Laboratory, University of New South Wales, Sydney, NSW, Australia.
Lee B; Biological Resources Imaging Laboratory, University of New South Wales, Sydney, NSW, Australia.
Suchowerska AK; Translational Neuroscience Facility and Department of Physiology, School of Biomedical Sciences, University of New South Wales, Sydney, NSW, Australia.
Housley GD; Translational Neuroscience Facility and Department of Physiology, School of Biomedical Sciences, University of New South Wales, Sydney, NSW, Australia.
Klugmann M; Translational Neuroscience Facility and Department of Physiology, School of Biomedical Sciences, University of New South Wales, Sydney, NSW, Australia.

Front Mol Neurosci ; 15: 1061257, 2022.

Article en En | MEDLINE | ID: mdl-36568275

Palabras clave

ASPA; Canavan disease; NAA (N-acetyl-L-aspartate); NAT8L; adeno-associated virus; aspartoacylase; gene therapy; leukodystrophy

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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Front Mol Neurosci Año: 2022 Tipo del documento: Article País de afiliación: Australia Pais de publicación: Suiza

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