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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Detalhes bibliográficos
Publicado no:Assay Drug Dev Technol
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2017
Assuntos:
Original Articles
Acesso em linha: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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