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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Detalhes bibliográficos
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
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2014
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Original Article
Acesso em linha: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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