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Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery

The reversibility of neuropathic lysosomal storage diseases, including MPS IIIA, is a major goal in therapeutic development, due to typically late diagnoses and a large population of untreated patients. We used self-complementary adeno-associated virus (scAAV) serotype 9 vector expressing human N-su...

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Detalles Bibliográficos
Autores principales:	Fu, Haiyan, Cataldi, Marcela P, Ware, Tierra A, Zaraspe, Kimberly, Meadows, Aaron S, Murrey, Darren A, McCarty, Douglas M
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2016
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4898406/ https://www.ncbi.nlm.nih.gov/pubmed/27331076 http://dx.doi.org/10.1038/mtm.2016.36

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