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

10.4231/NK5J-6T74

David H Thompson , Judith Storch

10/25/2022

2-Hydroxypropyl-β-cyclodextrin containing polyrotaxanes represent an attractive approach for treatment of a rare metabolic disorder Niemann-Pick Type C.

Chemistry Cholesterol Niemann-Pick β-CD polyrotaxanes