Corrective GUSB Transfer to the Canine Mucopolysaccharidosis VII Brain

Severe deficiency in lysosomal β-glucuronidase (β-glu) enzymatic activity results in mucopolysaccharidosis (MPS) VII, an orphan disease with symptoms often appearing in early childhood. Symptoms are variable, but many patients have multiple organ disorders including neurological defects. At the cell...

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Bibliographic Details
Main Authors:	Cubizolle, Aurelie, Serratrice, Nicolas, Skander, Nadia, Colle, Marie-Anne, Ibanes, Sandy, Gennetier, Aurelie, Bayo-Puxan, Neus, Mazouni, Khalil, Mennechet, Franck, Joussemet, Beatrice, Cherel, Yan, Lajat, Yaouen, Vite, Charles, Bernex, Florence, Kalatzis, Vasiliki, Haskins, Mark E, Kremer, Eric J
Format:	Artigo
Language:	Inglês
Published:	Nature Publishing Group 2014
Subjects:	Original Article
Online Access:	https://ncbi.nlm.nih.gov/pmc/articles/PMC3983960/ https://ncbi.nlm.nih.gov/pubmed/24343103 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.283
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Corrective GUSB Transfer to the Canine Mucopolysaccharidosis VII Brain

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