Micelle-like nanoparticles as carriers for DNA and siRNA.
Micelle-like nanoparticles as carriers for DNA and siRNA.
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DOI：
10.1021/mp5007213
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发表时间：
2015-02-02
影响因子：
4.9
通讯作者：
Torchilin VP
中科院分区：
文献类型：
--
作者：
Navarro G;Pan J;Torchilin VP
基因治疗是一种潜在的有效的疾病预防和治疗方法。然而，由于基因分子在体内的稳定性较差，因此需要与递送系统结合，以克服细胞外和细胞内屏障并允许进入作用位点。阳离子聚合物纳米颗粒是小干扰RNA（siRNA）和基于DNA的治疗剂的流行载体，其中有效和安全的递送是需要优化的重要因素。胶束样纳米颗粒（MNP）（半胶束，半聚合物纳米颗粒）可以通过将两种递送系统中的最佳者统一在一个单一载体中来克服这种阳离子载体的一些缺点。在这篇综述中，我们将讨论MNP的独特性质，包括自组装，缩合和保护核酸，改善细胞缔合和基因转染，以及低毒性可能有助于成功应用siRNA和DNA为基础的治疗进入临床。MNP的最新发展涉及增加刺激敏感功能以特异性地响应病理或外部施加的“触发物”（例如，温度、pH或酶催化、光或磁场）。最后，我们将概述MNP作为二合一载体用于同时递送不同药剂（小分子、成像剂）和核酸组合的用途。
Gene therapy represents a potential efficient approach of disease prevention and therapy. However, due to their poor in vivo stability, gene molecules need to be associated with delivery systems to overcome extracellular and intracellular barriers and allow access to the site of action. Cationic polymeric nanoparticles are popular carriers for small interfering RNA (siRNA) and DNA-based therapeutics for which efficient and safe delivery are important factors that need to be optimized. Micelle-like nanoparticles (MNP) (half micelles, half polymeric nanoparticles) can overcome some of the disadvantages of such cationic carriers by unifying in one single carrier the best of both delivery systems. In this review, we will discuss how the unique properties of MNP including self-assembly, condensation and protection of nucleic acids, improved cell association and gene transfection, and low toxicity may contribute to the successful application of siRNA- and DNA-based therapeutics into the clinic. Recent developments of MNP involving the addition of stimulus-sensitive functions to respond specifically to pathological or externally applied “triggers” (e.g., temperature, pH or enzymatic catalysis, light, or magnetic fields) will be discussed. Finally, we will overview the use of MNP as two-in-one carriers for the simultaneous delivery of different agents (small molecules, imaging agents) and nucleic acid combinations.
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