Centre de Recherche en Cancérologie de Marseille, Inserm, UMR1068, 13009 Marseille, France; Institut Paoli-Calmettes, 13009 Marseille, France; Aix-Marseille Université, 13284 Marseille, France; CNRS, UMR7258, 13009 Marseille, France.
Aix-Marseille Université, Centre Interdisciplinaire de Nanoscience de Marseille, CNRS UMR 7325, 163, avenue de Luminy, 13288 Marseille, France; College of Chemistry and Molecular Sciences, Wuhan University, 430072 Wuhan, P. R. China.
Biotechnol Adv. 2014 Jul-Aug;32(4):844-52. doi: 10.1016/j.biotechadv.2013.08.001. Epub 2013 Aug 9.
RNAi-based nucleic acid molecules have attracted considerable attention as compelling therapeutics providing safe and competent delivery systems are available. Dendrimers are emerging as appealing nanocarriers for nucleic acid delivery thanks to their unique well-defined architecture and the resulting cooperativity and multivalency confined within a nanostructure. The present review offers a brief overview of the structurally flexible triethanolamine-core poly(amidoamine) (PAMAM) dendrimers developed in our group as nanovectors for the delivery of RNAi therapeutics. Their excellent activity for delivering different RNAi therapeutics in various disease models in vitro and in vivo will be highlighted here.
基于 RNAi 的核酸分子作为一种有吸引力的治疗方法引起了相当大的关注，因为现在有安全有效的输送系统。树状聚合物由于其独特的明确结构以及纳米结构内受限的协同作用和多价性，成为有吸引力的核酸输送纳米载体。本综述简要概述了我们小组开发的作为 RNAi 治疗药物输送的纳米载体的结构灵活的三乙醇胺核聚酰胺-胺（PAMAM）树状聚合物。这里将重点介绍它们在体外和体内各种疾病模型中递送不同 RNAi 治疗药物的优异活性。
