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

The leukodystrophy Canavan disease is a fatal white matter disorder caused by loss-of-function mutations of the aspartoacylase-encoding ASPA gene. There are no effective treatments available and experimental gene therapy trials have failed to provide sufficient amelioration from Canavan disease symp...

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Detalles Bibliográficos
Autores principales:	Fröhlich, Dominik, Kalotay, Elizabeth, von Jonquieres, Georg, Bongers, Andre, Lee, Brendan, Suchowerska, Alexandra K., Housley, Gary D., Klugmann, Matthias
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2022
Materias:	Molecular Neuroscience
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9772617/ https://www.ncbi.nlm.nih.gov/pubmed/36568275 http://dx.doi.org/10.3389/fnmol.2022.1061257

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