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Article

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

2021-04-09

Abstract excerpt

Niemann-Pick type C (NPC) 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 and degeneration of specific cells, notably neurons in the central nervous system (CNS). 2-hydroxypropyl-beta-...

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Literature Corpus work
34f24e64-5b44-5e83-aa0c-2466655906ab
DOI
10.1101/2021.04.08.438990
Open publication

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Glial contribution to cyclodextrin-mediated reversal of cholesterol accumulation in murine NPC1-deficient neurons <i>in vivo</i>DOI 10.1101/2021.04.08.438990
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