Enzyme replacement in a canine model of Hurler syndrome.

The Hurler syndrome (alpha-L-iduronidase deficiency disease) is a severe lysosomal storage disorder that is potentially amenable to enzyme-replacement therapy. Availability of a canine model of the disease and a sufficient supply of corrective enzyme have permitted a therapeutic trial lasting 3 mo....

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Bibliografiske detaljer

Udgivet i:	Proc Natl Acad Sci U S A
Principais autores:	Shull, R M, Kakkis, E D, McEntee, M F, Kania, S A, Jonas, A J, Neufeld, E F
Format:	Artigo
Sprog:	Inglês
Udgivet:	National Academy of Sciences 1994
Fag:	Research Article
Online adgang:	https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC45555/ https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/7809150/ https://ncbi.nlm.nih.govhttps://doi.org/10.1073/pnas.91.26.12937
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