Cyclodextrin‐Based Macromolecular Systems as Cholesterol‐Mopping Therapeutic Agents in Niemann–Pick Disease Type C,Macromolecular Rapid Communications

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Cyclodextrin‐Based Macromolecular Systems as Cholesterol‐Mopping Therapeutic Agents in Niemann–Pick Disease Type C
Macromolecular Rapid Communications ( IF 4.2 ) Pub Date : 2018-09-14 , DOI: 10.1002/marc.201800557
Antonino Puglisi ₁ , Yusuf Yagci ₁

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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.

中文翻译：

基于环糊精的大分子系统作为C型尼曼匹克病的胆固醇清除剂

在过去的十年中，环糊精（CDs）作为治疗稀有遗传性尼曼-皮克C型疾病（NPC）的潜在治疗手段而受到了广泛关注。然而，单体形式的寡糖遭受严重的副作用，特别是从药代动力学和生物分布的观点来看。基于CD的大分子系统有望克服这些局限性，并可能为减少NPC中的胆固醇积累提供改进的治疗方法。在本文中，总结了制备含CD的聚合物作为NPC中的胆固醇清除治疗剂的最新进展和合成策略。

更新日期：2018-09-14

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