Long Circulating Enzyme Replacement Therapy Rescues Bone Pathology in Mucopolysaccharidosis VII Murine Model

Mucopolysaccharidosis (MPS) type VII is a lysosomal storage disease caused by deficiency of the lysosomal enzyme β-glucuronidase (GUS), leading to accumulation of glycosaminoglycans (GAGs). Enzyme replacement therapy (ERT) effectively clears GAG storage in the viscera. Recent studies showed that a c...

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Detalhes bibliográficos
Main Authors: Rowan, Daniel J., Tomatsu, Shunji, Grubb, Jeffrey H., Haupt, Bisong, Montaño, Adriana M., Oikawa, Hirotaka, Sosa, Catalina, Chen, Anping, Sly, William S.
Formato: Artigo
Idioma:Inglês
Publicado em: 2012
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3444660/
https://ncbi.nlm.nih.gov/pubmed/22902520
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2012.07.002
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