Intracisternal Cyclodextrin Prevents Cerebellar Dysfunction and Purkinje Cell Death in Feline Niemann-Pick type C1 disease

Niemann-Pick type C1 (NPC) disease is a lysosomal storage disease caused by mutations in the NPC1 gene, leading to an increase in unesterified cholesterol and several sphingolipids, and resulting in hepatic disease and progressive neurological disease. Whereas subcutaneous administration of the phar...

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Bibliographic Details
Published in:Sci Transl Med
Main Authors: Vite, C. H., Bagel, J. H., Swain, G. P., Prociuk, M., Sikora, T. U., Stein, V. M., O’Donnell, P., Ruane, T., Ward, S., Crooks, A., Li, S., Mauldin, E., Stellar, S., De Meulder, M., Kao, M. L., Ory, D. S., Davidson, C., Vanier, M. T., Walkley, S. U.
Format: Artigo
Language:Inglês
Published: 2015
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Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC4415615/
https://ncbi.nlm.nih.gov/pubmed/25717099
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.3010101
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