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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書誌詳細
主要な著者: 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
フォーマット: Artigo
言語:Inglês
出版事項: Nature Publishing Group 2012
主題:
Original Article
オンライン･アクセス: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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