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Glycosylation-independent targeting enhances enzyme delivery to lysosomes and decreases storage in mucopolysaccharidosis type VII mice

Enzyme-replacement therapy is an established means of treating lysosomal storage diseases. Infused therapeutic enzymes are targeted to lysosomes of affected cells by interactions with cell-surface receptors that recognize carbohydrate moieties, such as mannose and mannose 6-phosphate, on the enzymes...

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Bibliografiske detaljer
Main Authors: LeBowitz, Jonathan H., Grubb, Jeffrey H., Maga, John A., Schmiel, Deborah H., Vogler, Carole, Sly, William S.
Format: Artigo
Sprog:Inglês
Udgivet: National Academy of Sciences 2004
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC365748/
https://ncbi.nlm.nih.gov/pubmed/14976248
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0308728100
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