Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII

Mucopolysaccharidosis VII (MPS VII) is a lysosomal storage disease arising from mutations in β-d-glucuronidase (GUSB), which results in glycosaminoglycan (GAG) accumulation and a variety of clinical manifestations including neurological disease. Herein, MPS VII dogs were injected intravenously (i.v....

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Veröffentlicht in:Mol Ther
Hauptverfasser: Gurda, Brittney L, De Guilhem De Lataillade, Adrien, Bell, Peter, Zhu, Yanqing, Yu, Hongwei, Wang, Ping, Bagel, Jessica, Vite, Charles H, Sikora, Tracey, Hinderer, Christian, Calcedo, Roberto, Yox, Alexander D, Steet, Richard A, Ruane, Therese, O'Donnell, Patricia, Gao, Guangping, Wilson, James M, Casal, Margret, Ponder, Katherine P, Haskins, Mark E
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2016
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Original Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4817811/
https://ncbi.nlm.nih.gov/pubmed/26447927
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.189
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