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Cyclodextrin alleviates neuronal storage of cholesterol in Niemann-Pick C disease without evidence of detectable blood-brain barrier permeability

Niemann Pick type C disease is an inherited autosomal recessive disorder characterised by the accumulation of unesterified cholesterol and sphingolipids within the endosomal/lysosomal compartments. It has been observed that the administration of hydroxypropyl-β-cyclodextrin (HPBCD) delays onset of c...

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Bibliografiska uppgifter
Huvudupphovsmän: Pontikis, Charles C., Davidson, Cristin D., Walkley, Steven U., Platt, Frances M., Begley, David J
Materialtyp: Artigo
Språk:Inglês
Publicerad: 2013
Ämnen:
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC3929395/
https://ncbi.nlm.nih.gov/pubmed/23412751
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-012-9583-x
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