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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Bibliographic Details
Main Authors: 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
Format: Artigo
Language:Inglês
Published: Nature Publishing Group 2012
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Online Access: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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