Analytical Characterization of Methyl-β-Cyclodextrin for Pharmacological Activity to Reduce Lysosomal Cholesterol Accumulation in Niemann-Pick Disease Type C1 Cells

Methyl-β-cyclodextrin (MβCD) reduces lysosomal cholesterol accumulation in Niemann-Pick disease type C1 (NPC1) patient fibroblasts. However, the pharmacological activity of MβCD reported by different laboratories varies. To determine the potential causes of this variation, we analyzed the mass spect...

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Bibliografische gegevens
Gepubliceerd in:Assay Drug Dev Technol
Hoofdauteurs: Li, Rong, Hao, Jon, Fujiwara, Hideji, Xu, Miao, Yang, Shu, Dai, Sheng, Long, Yan, Swaroop, Manju, Li, Changhui, Vu, Mylinh, Marugan, Juan J., Ory, Daniel S., Zheng, Wei
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Mary Ann Liebert, Inc. 2017
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Original Articles
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5510037/
https://ncbi.nlm.nih.gov/pubmed/28631941
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/adt.2017.774
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