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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Enregistré dans:
Détails bibliographiques
Publié dans:J Clin Invest
Auteurs principaux: Sands, M S, Vogler, C, Torrey, A, Levy, B, Gwynn, B, Grubb, J, Sly, W S, Birkenmeier, E H
Format: Artigo
Langue:Inglês
Publié: American Society for Clinical Investigation 1997
Sujets:
Research Article
Accès en ligne:https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC507979/
https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/9120003/
https://ncbi.nlm.nih.govhttps://doi.org/10.1172/JCI119322
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