Murine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation.

We demonstrated previously that short term administration of recombinant beta-glucuronidase to newborn mice with mucopolysaccharidosis type VII reduced lysosomal storage in many tissues. Lysosomal storage accumulated gradually after cessation of enzyme replacement therapy. Mice alive at 1 yr of age...

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Detaylı Bibliyografya
Asıl Yazarlar: Sands, M S, Vogler, C, Torrey, A, Levy, B, Gwynn, B, Grubb, J, Sly, W S, Birkenmeier, E H
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: 1997
Konular:
Research Article
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC507979/
https://ncbi.nlm.nih.gov/pubmed/9120003
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