Correction of Neurological Disease of Mucopolysaccharidosis IIIB in Adult Mice by rAAV9 Trans-Blood–Brain Barrier Gene Delivery

The greatest challenge in developing therapies for mucopolysaccharidosis (MPS) IIIB is to achieve efficient central nervous system (CNS) delivery across the blood–brain barrier (BBB). In this study, we used the novel ability of adeno-associated virus serotype 9 (AAV9) to cross the BBB from the vascu...

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Autores principales: Fu, Haiyan, DiRosario, Julianne, Killedar, Smruti, Zaraspe, Kimberly, McCarty, Douglas M
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group 2011
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Original Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3129800/
https://ncbi.nlm.nih.gov/pubmed/21386820
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.34
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