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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Bibliografski detalji
Glavni autori: 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
Jezik:Inglês
Izdano: Nature Publishing Group 2014
Teme:
Original Article
Online pristup: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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