Glial contribution to cyclodextrin-mediated reversal of cholesterol accumulation in murine NPC1-deficient neurons in vivo

Glial contribution to cyclodextrin-mediated reversal of cholesterol accumulation in murine NPC1-deficient neurons in vivo

Niemann-Pick type C disease is a rare and fatal lysosomal storage disorder presenting severe neurovisceral symptoms. Disease-causing mutations in genes encoding either NPC1 or NPC2 protein provoke accumulation of cholesterol and other lipids in specific structures of the endosomal-lysosomal system a...

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Bibliographic Details
Main Authors: Amélie Barthelemy, Valérie Demais, Izabela-Cristina Stancu, Eugeniu Vasile, Tom Houben, Michael Reber, Valentina Pallottini, Martine Perraut, Sophie Reibel, Frank W. Pfrieger
Format: Article
Language:English
Published: Elsevier 2021-10-01
Series:Neurobiology of Disease
Subjects:
Lysosome
Intravitreal
Cyclodextrin
Inherited metabolic disease
Ganglion cells
Amacrine cells
Online Access:http://www.sciencedirect.com/science/article/pii/S0969996121002187

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http://www.sciencedirect.com/science/article/pii/S0969996121002187

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