树形分子在siRNA递送载体中的应用

Application of dendrimer-based siRNA delivery systems

摘要: 基于小干扰核酸分子(siRNA)的RNA干扰技术作为极具潜力的治疗策略吸引了越来越多的学术界和工业界的关注，如何实现siRNA的临床转化日渐成为生物医药领域的研究热点。然而，转化成功与否很大程度上依赖于安全高效的siRNA递送系统。树形分子作为一种新型的大分子，具有精确可控的化学结构、可供修饰的末端基团以及纳米尺寸等独特的物理化学性质，广泛应用于siRNA的递送。本文归纳和总结了不同种类的树形分子在siRNA递送领域的研究进展，为后续树形分子用于siRNA递送的研究提供思路。

Abstract: Small interfering RNA-based RNA interference(RNAi)is an emerging treatment which has attracted more and more attention from both academia and industry. Studies on clinical translation of siRNA are popular in biomedical field, where safe and efficient carriers are necessary. As novel drug/gene vectors, dendrimers are widely used in siRNA delivery due to their unique and precise structure, multiple terminal groups and nano-sized chemophysical properties. Here, we present an overall view of current studies on dendrimer-based siRNA delivery systems, with the aim to provide an understanding of future siRNA therapeutics which could be used in clinic.