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Targeting of the CNS in MPS-IH Using a Nonviral Transferrin-α-l-iduronidase Fusion Gene Product

Mucopolysaccharidosis type I (Hurler syndrome) is caused by a deficiency of the enzyme α-l-iduronidase (IDUA), and is characterized by widespread lysosomal glycosaminoglycan (GAG) accumulation. Successful treatment of central nervous system (CNS) diseases is limited by the presence of the blood–brai...

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Detalles Bibliográficos
Main Authors: Osborn, Mark J, McElmurry, Ron T, Peacock, Brandon, Tolar, Jakub, Blazar, Bruce R
Formato: Artigo
Idioma:Inglês
Publicado: 2008
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Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC2574880/
https://ncbi.nlm.nih.gov/pubmed/18523448
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2008.119
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