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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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4817811/
https://www.ncbi.nlm.nih.gov/pubmed/26447927
http://dx.doi.org/10.1038/mt.2015.189

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