Cyclodextrin overcomes the transport defect in nearly every organ of NPC1 mice leading to excretion of sequestered cholesterol as bile acid.

@article{Liu2010CyclodextrinOT,
  title={Cyclodextrin overcomes the transport defect in nearly every organ of NPC1 mice leading to excretion of sequestered cholesterol as bile acid.},
  author={Benny Liu and Charina M. Ramirez and Anna M Miller and Joyce J. Repa and Stephen D. Turley and John M. Dietschy},
  journal={Journal of lipid research},
  year={2010},
  volume={51 5},
  pages={933-44}
}
A mutation in NPC1 leads to sequestration of unesterified cholesterol in the late endosomal/lysosomal compartment of every cell culminating in the development of pulmonary, hepatic, and neurodegenerative disease. Acute administration of 2-hydroxypropyl-beta-cyclodextrin (CYCLO) rapidly overcomes this transport defect in both the 7-day-old pup and 49-day-old mature npc1(-/-) mouse, even though this compound is cleared from the body and plasma six times faster in the mature mouse than in the… CONTINUE READING