Liver Production of Sulfamidase Reverses Peripheral and Ameliorates CNS Pathology in Mucopolysaccharidosis IIIA Mice

Mucopolysaccharidosis type IIIA (MPSIIIA) is an inherited lysosomal storage disease caused by deficiency of sulfamidase, resulting in accumulation of the glycosaminoglycan (GAG) heparan sulfate. It is characterized by severe progressive neurodegeneration, together with somatic alterations, which lea...

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Autori principali: Ruzo, Albert, Garcia, Miquel, Ribera, Albert, Villacampa, Pilar, Haurigot, Virginia, Marcó, Sara, Ayuso, Eduard, Anguela, Xavier M, Roca, Carles, Agudo, Judith, Ramos, David, Ruberte, Jesús, Bosch, Fatima
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2012
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3278559/
https://ncbi.nlm.nih.gov/pubmed/22008915
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.220
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