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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...
Autores principales: | 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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Mary Ann Liebert, Inc.
2017
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5510037/ https://www.ncbi.nlm.nih.gov/pubmed/28631941 http://dx.doi.org/10.1089/adt.2017.774 |
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