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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Bibliografische gegevens
Hoofdauteurs: 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
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Nature Publishing Group 2012
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Original Article
Online toegang: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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