Weekly Cyclodextrin Administration Normalizes Cholesterol Metabolism in Nearly Every Organ of the Niemann-Pick Type C1 Mouse and Markedly Prolongs Life

Murine Model of Niemann-Pick C Disease: Mutation in a Cholesterol Homeostasis Gene

Stacie K. Loftus, Jill A. Morris, Eugene D. Carstea et al. · Science · 1997 · 807 citations

Life Sciences, Genetics, Molecular Biology +17

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

Cristin Davidson, Nafeeza Ali, Matthew C. Micsenyi et al. · PLoS ONE · 2009 · 451 citations · Full text

Life Sciences, Neuroscience, Pharmacology +17

Reversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the <i>npc1</i> ^−/− mouse

Benny Liu, Stephen D. Turley, Dennis K. Burns et al. · Proceedings of the National Academy of Sciences · 2009 · 380 citations · Full text

Life Sciences, Biochemistry, Cell Biology +17

Neurons in Niemann-Pick Disease Type C Accumulate Gangliosides as Well as Unesterified Cholesterol and Undergo Dendritic and Axonal Alterations

Mark Zervas, Kostantin Dobrenis, Steven U. Walkley · Journal of Neuropathology & Experimental Neurology · 2001 · 267 citations · Full text

Life Sciences, Neurobiology, Neuroscience +17

Cyclodextrin overcomes the transport defect in nearly every organ of NPC1 mice leading to excretion of sequestered cholesterol as bile acid

Benny Liu, Charina M. Ramirez, Anna M. Miller et al. · Journal of Lipid Research · 2009 · 173 citations · Full text

Life Sciences, Biochemistry, Pharmacology +17