In this focus review, the basic principle of stimuli-cleavable polyrotaxane (PRX) comprising many cyclic molecules threaded onto a linear polymer chain capped with bulky stopper molecules via cleavable linkers are described. Additionally, recent progress in the use of β-cyclodextrin-threaded stimuli-cleavable PRXs as a therapeutic agent for Niemann–Pick-type C (NPC) disease is described. The lysosomal release of threaded β-cyclodextrins from PRXs leads to the formation of an inclusion complex with cholesterols accumulated in NPC disease, promoting extracellular excretion of cholesterols.
- Atsushi Tamura
- Nobuhiko Yui
