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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Опубликовано в: :Mol Ther
Главные авторы: 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
Формат: Artigo
Язык:Inglês
Опубликовано: Nature Publishing Group 2016
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Original Article
Online-ссылка: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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