Numerous transcriptional alterations in liver persist after short-term enzyme-replacement therapy in a murine model of mucopolysaccharidosis type VII.

The lysosomal storage disease MPS VII (mucopolysaccharidosis type VII) is caused by a deficiency in beta-glucuronidase activity, and results in the accumulation of partially degraded glycosaminoglycans in many cell types. Although MPS VII is a simple monogenetic disorder, the clinical presentation i...

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Autores principales: Woloszynek, Josh C, Roberts, Marie, Coleman, Trey, Vogler, Carole, Sly, William, Semenkovich, Clay F, Sands, Mark S
Formato: Artigo
Lenguaje:Inglês
Publicado: 2004
Materias:
Research Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC1224072/
https://ncbi.nlm.nih.gov/pubmed/14705966
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1042/BJ20031048
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