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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...
Gorde:
| Egile Nagusiak: | , , , , , , , , , , , , |
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| Formatua: | Artigo |
| Hizkuntza: | Inglês |
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Nature Publishing Group
2012
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| Sarrera elektronikoa: | 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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