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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Identifiers and source
- Literature Corpus work
- 34f24e64-5b44-5e83-aa0c-2466655906ab
- DOI
- 10.1101/2021.04.08.438990
