PEG-lipid micelles enable cholesterol efflux in Niemann-Pick Type C1 disease-based lysosomal storage disorder

2-Hydroxy-propyl-β-cyclodextrin (HPβCD), a cholesterol scavenger, is currently undergoing Phase 2b/3 clinical trial for treatment of Niemann Pick Type C-1 (NPC1), a fatal neurodegenerative disorder that stems from abnormal cholesterol accumulation in the endo/lysosomes. Unfortunately, the extremely...

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Bibliographic Details
Published in:Sci Rep
Main Authors: Brown, Anna, Patel, Siddharth, Ward, Carl, Lorenz, Anna, Ortiz, Mauren, DuRoss, Allison, Wieghardt, Fabian, Esch, Amanda, Otten, Elsje G., Heiser, Laura M., Korolchuk, Viktor I., Sun, Conroy, Sarkar, Sovan, Sahay, Gaurav
Format: Artigo
Language:Inglês
Published: Nature Publishing Group 2016
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Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC5004151/
https://ncbi.nlm.nih.gov/pubmed/27572704
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/srep31750
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