Regenerative capacity of the olfactory epithelium in Niemann-Pick disease type C1 after a substrate reduction therapy and cyclodextrin

Anja Meyer, Susann Lehmann, Oliver Schmitt, Andreas Wree & Martin Witt
Introduction: Niemann-Pick disease type C1 (NPC1) is a fatal neurovisceral lysosomal lipid storage disorder. The mutation of the NPC1 gene affects the cholesterol homeostasis and the transport of cholesterol and glycosphingolipids from late endosomes/ lysosomes to the endoplasmic reticulum resulting[for full text, please go to the a.m. URL]
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