التفاصيل البيبلوغرافية
| العنوان: |
Analytical Characterization of Methyl-β-Cyclodextrin for Pharmacological Activity to Reduce Lysosomal Cholesterol Accumulation in Niemann-Pick Disease Type C1 Cells. |
| المؤلفون: |
Rong Li, Jon Hao, Hideji Fujiwara, Miao Xu, Shu Yang, Sheng Dai, Yan Long, Swaroop, Manju, Changhui Li, Mylinh Vu, Marugan, Juan J., Ory, Daniel S., Wei Zheng |
| المصدر: |
Assay & Drug Development Technologies; May/Jun2017, Vol. 15 Issue 4, p154-166, 13p |
| مصطلحات موضوعية: |
CYCLODEXTRINS, LYSOSOMAL storage diseases, NIEMANN-Pick diseases, NON-langerhans-cell histiocytosis |
| مستخلص: |
Methyl-b-cyclodextrin (MbCD) reduces lysosomal cholesterol accumulation in Niemann-Pick disease type C1 (NPC1) patient fibroblasts. However, the pharmacological activity of MbCD reported by different laboratories varies. To determine the potential causes of this variation, we analyzed the mass spectrum characteristics, pharmacological activity of three preparations of MbCDs, and the protein expression profiles of NPC1 patient fibroblasts after treatment with different sources of MbCDs. Our data revealed varied mass spectrum profiles and pharmacological activities on the reduction of lysosomal cholesterol accumulation in NPC1 fibroblasts for these three preparations of MbCDs obtained from different batches and different sources. Furthermore, a proteomic analysis showed the differences of these three MbCD preparations on amelioration of dysregulated protein expression levels in NPC1 cells. The results demonstrate the importance of prescreening of different cyclodextrin preparations before use as a therapeutic agent. A combination of mass spectrum analysis, measurement of pharmacological activity, and proteomic profiling provides an effective analytical procedure for characterization of cyclodextrins for therapeutic applications. [ABSTRACT FROM AUTHOR] |
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| قاعدة البيانات: |
Complementary Index |
