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化学进展  2014 

基于环糊精构建的基因载体进展

DOI: 10.7536/PC130632, PP. 375-384

Keywords: 环糊精,超分子,自组装,基因载体

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Abstract:

安全有效的基因载体对于基因治疗有着重要的潜在价值。相对于病毒性基因载体,化学合成的载体具有低免疫原性,易于大规模生产和生产成本较低的特性,因而受到越来越多的关注,但是非病毒基因载体在转染效率和选择性方面有一定的限制性,当前的主要研究工作集中在这两方面。基于环糊精构建的基因载体,可以有效地提供基因载体的立体构象和功能性的选择性。作为FDA批准的生物材料,环糊精具有无毒和生物降解性,其不但可以保护基因,避免在体内降解,同时有助于通过细胞膜,进入细胞内达到基因转染的作用。环糊精具有大量可修饰的羟基基团,因此对环糊精修饰不但可以通过主客体作用构建超分子体系,并且可以作为多官能团核形成星状高分子,被广泛应用于制备低毒、可降解、靶向性和高效率的转基因载体。目前,环糊精修饰的非病毒正离子载体转移siRNA,已经成功进行了色素瘤的临床试验,取得了很好的治疗效果,表明了环糊精非病毒载体的巨大应用前景。本文对基于环糊精基因载体的最新研究进展进行了综述,详细介绍了基于环糊精的超分子自组装构建的聚轮烷型、侧链分子识别型的基因载体,以及基于环糊精多羟基构型而构建的星型聚合物基因载体和树枝状基因载体,并对环糊精基因载体的优越性和未来应用做了相应的介绍。

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