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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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Fu, Haiyan, Cataldi, Marcela P, Ware, Tierra A, Zaraspe, Kimberly, Meadows, Aaron S, Murrey, Darren A, McCarty, Douglas M
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4898406/
https://ncbi.nlm.nih.gov/pubmed/27331076
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2016.36
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