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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Vydáno v:Mol Ther
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2016
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Original Article
On-line přístup: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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