Cholesterol homeostatic responses provide biomarkers for monitoring treatment for the neurodegenerative disease Niemann–Pick C1 (NPC1)

Niemann–Pick C1 (NPC1) disease is a rare, neurodegenerative lysosomal cholesterol storage disorder, typified by progressive cognitive and motor function impairment. Affected individuals usually succumb to the disease in adolescence. 2-Hydroxypropyl-β-cyclodextrin (HP-β-CD) has emerged as a promising...

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Autores principales: Tortelli, Brett, Fujiwara, Hideji, Bagel, Jessica H., Zhang, Jessie, Sidhu, Rohini, Jiang, Xuntian, Yanjanin, Nicole M., Shankar, Roopa Kanakatti, Carillo-Carasco, Nuria, Heiss, John, Ottinger, Elizabeth, Porter, Forbes D., Schaffer, Jean E., Vite, Charles H., Ory, Daniel S.
Formato: Artigo
Lenguaje:Inglês
Publicado: Oxford University Press 2014
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Articles
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC4204776/
https://ncbi.nlm.nih.gov/pubmed/24964810
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddu331
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