FTY720/fingolimod increases NPC1 and NPC2 expression and reduces cholesterol and sphingolipid accumulation in Niemann-Pick type C mutant fibroblasts

Niemann-Pick type C (NPC) disease is a fatal neurodegenerative disorder caused by mutations in NPC1 or NPC2 with decreased functions leading to lysosomal accumulation of cholesterol and sphingolipids. FTY720/fingolimod, used for treatment of multiple sclerosis, is phosphorylated by nuclear sphingosi...

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Veröffentlicht in:FASEB J
Hauptverfasser: Newton, Jason, Hait, Nitai C., Maceyka, Michael, Colaco, Alexandria, Maczis, Melissa, Wassif, Christopher A., Cougnoux, Antony, Porter, Forbes D., Milstien, Sheldon, Platt, Nicholas, Platt, Frances M., Spiegel, Sarah
Format: Artigo
Sprache:Inglês
Veröffentlicht: Federation of American Societies for Experimental Biology 2017
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Research
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5349795/
https://ncbi.nlm.nih.gov/pubmed/28082351
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1096/fj.201601041R
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