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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Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2014
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Original Article
On-line přístup: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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