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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Bibliografski detalji
Izdano u:Sci Transl Med
Glavni autori: 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
Jezik:Inglês
Izdano: 2015
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Article
Online pristup: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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