Chronic Cyclodextrin Treatment of Murine Niemann-Pick C Disease Ameliorates Neuronal Cholesterol and Glycosphingolipid Storage and Disease Progression

BACKGROUND: Niemann-Pick type C (NPC) disease is a fatal neurodegenerative disorder caused most commonly by a defect in the NPC1 protein and characterized by widespread intracellular accumulation of unesterified cholesterol and glycosphingolipids (GSLs). While current treatment therapies are limited...

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Autors principals: Davidson, Cristin D., Ali, Nafeeza F., Micsenyi, Matthew C., Stephney, Gloria, Renault, Sophie, Dobrenis, Kostantin, Ory, Daniel S., Vanier, Marie T., Walkley, Steven U.
Format: Artigo
Idioma:Inglês
Publicat: Public Library of Science 2009
Matèries:
Research Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC2736622/
https://ncbi.nlm.nih.gov/pubmed/19750228
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0006951
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