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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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Detalles Bibliográficos
Publicado en:Proc Natl Acad Sci U S A
Autores principales: Shull, R M, Kakkis, E D, McEntee, M F, Kania, S A, Jonas, A J, Neufeld, E F
Formato: Artigo
Lenguaje:Inglês
Publicado: National Academy of Sciences 1994
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Acceso en línea: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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