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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Bibliographic Details
Main Authors: Newton, J, Hait, N, Maceyka, M, Colaco, A, Maczis, M, Wassif, C, Cougnoux, A, Porter, F, Milstien, S, Platt, N, Platt, F, Spiegel, S
Format: Journal article
Language:English
Published: Federation of American Societies for Experimental Biology 2017