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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Dades bibliogràfiques
Publicat a:Assay Drug Dev Technol
Autors principals: 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
Format: Artigo
Idioma:Inglês
Publicat: Mary Ann Liebert, Inc. 2017
Matèries:
Original Articles
Accés en línia: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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