Cyclodextrins in the treatment of a mouse model of Niemann-Pick C disease
β Scribed by Fernando Camargo; Robert P. Erickson; William S. Garver; G.Showkat Hossain; Peter N. Carbone; Randall A. Heidenreich; James Blanchard
- Book ID
- 117173243
- Publisher
- Elsevier Science
- Year
- 2001
- Tongue
- English
- Weight
- 138 KB
- Volume
- 70
- Category
- Article
- ISSN
- 0024-3205
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## Abstract The BALB/c mouse model of NiemannβPick type C (NPC) disease exhibits neuropathological similarities to the human condition. There is an ageβrelated cerebral atrophy, demyelination of the corpus callosum, and degeneration of cerebellar Purkinje cells in the NPC mouse. In human NPC, many
## Abstract NiemannβPick disease type C (NPC) is a progressive neurodegenerative disorder characterized by accumulation of free cholesterol in lysosomes, mainly due to a mutation in the NPC1 gene. The pathophysiological basis of the neural disorders in NPC, however, is not well understood. We found