Cyclodextrin-Based Macromolecular Systems as Cholesterol-Mopping Therapeutic Agents in Niemann-Pick Disease Type C

Abstract

Over the last decade, cyclodextrins (CDs) have gained considerable attention as a potential therapeutic intervention in the treatment of the rare genetic condition Niemann-Pick type C disease (NPC). However, the oligosaccharide in its monomeric form suffers from serious side effects, especially from a pharmacokinetic and biodistribution standpoint. CD-based macromolecular systems hold great promise to overcome such limitations and might provide an improved therapeutic approach in reducing cholesterol accumulation in NPC. In the present article, the latest developments and synthetic strategies in the preparation of CD-containing polymers as cholesterol-mopping therapeutic agents in NPC are summarized.

Keywords: Niemann-Pick type C; cholesterol; cyclodextrins; lysosomes; polymers.