Structure-function relationships of cholesterol mobilization from the endo-lysosome compartment of NPC1-deficient human cells by β-CD polyrotaxanes.

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...

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Bibliographic Details
Main Authors: Shayak Samaddar, Debosreeta Bose, Bradley P Loren, Joseph L Skulsky, Olga Ilnytska, Zachary J Struzik, Judith Storch, David H Thompson
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2022-01-01
Series:PLoS ONE
Online Access:https://doi.org/10.1371/journal.pone.0268613

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