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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Bibliografische gegevens
Gepubliceerd in:Sci Transl Med
Hoofdauteurs: 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.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2015
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Article
Online toegang: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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