Structure-function relationships of cholesterol mobilization from the endo-lysosome compartment of NPC1-deficient human cells by β-CD polyrotaxanes.
Niemann-Pick Type C is a rare metabolic disorder characterized by the cellular accumulation of cholesterol within endosomal and lysosomal compartments. 2-Hydroxypropyl-β-cyclodextrin (HP-β-CD) containing polyrotaxanes represent an attractive approach for treating this disease due to their ability to...
Main Authors: | Shayak Samaddar, Debosreeta Bose, Bradley P Loren, Joseph L Skulsky, Olga Ilnytska, Zachary J Struzik, Judith Storch, David H Thompson |
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Format: | Article |
Language: | English |
Published: |
Public Library of Science (PLoS)
2022-01-01
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Series: | PLoS ONE |
Online Access: | https://doi.org/10.1371/journal.pone.0268613 |
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