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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...

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Detalles Bibliográficos
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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc. 2017
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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