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Mannose 6-Phosphate Receptor–mediated Transport of Sulfamidase Across the Blood–brain Barrier in the Newborn Mouse

Mucopolysaccharidosis type IIIA (MPS IIIA), which is a lysosomal storage disorder (LSD) caused by inherited deficiency of sulfamidase, is characterized by severe, progressive central nervous system (CNS) dysfunction. Enzyme replacement therapy (ERT) to treat CNS storage is challenging, because the a...

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Bibliografiset tiedot
Päätekijät: Urayama, Akihiko, Grubb, Jeffrey H, Sly, William S, Banks, William A
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: 2008
Aiheet:
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC2908959/
https://ncbi.nlm.nih.gov/pubmed/18443601
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2008.84
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