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在应用 RNA 纳米技术的治疗学方面的独特性、优势、挑战、解决方案和展望。

Uniqueness, advantages, challenges, solutions, and perspectives in therapeutics applying RNA nanotechnology.

机构信息

Nanobiotechnology Center, Markey Cancer Center and Department of Pharmaceutical Sciences, University of Kentucky, Lexington, Kentucky 40536, USA.

出版信息

Nucleic Acid Ther. 2012 Aug;22(4):226-45. doi: 10.1089/nat.2012.0350.

Abstract

The field of RNA nanotechnology is rapidly emerging. RNA can be manipulated with the simplicity characteristic of DNA to produce nanoparticles with a diversity of quaternary structures by self-assembly. Additionally RNA is tremendously versatile in its function and some RNA molecules display catalytic activities much like proteins. Thus, RNA has the advantage of both worlds. However, the instability of RNA has made many scientists flinch away from RNA nanotechnology. Other concerns that have deterred the progress of RNA therapeutics include the induction of interferons, stimulation of cytokines, and activation of other immune systems, as well as short pharmacokinetic profiles in vivo. This review will provide some solutions and perspectives on the chemical and thermodynamic stability, in vivo half-life and biodistribution, yield and production cost, in vivo toxicity and side effect, specific delivery and targeting, as well as endosomal trapping and escape.

摘要

RNA 纳米技术领域正在迅速兴起。RNA 可以像 DNA 一样通过自组装来操纵,产生具有多种四级结构的纳米颗粒。此外,RNA 在功能上具有极大的多样性,一些 RNA 分子表现出类似于蛋白质的催化活性。因此,RNA 兼具两者的优势。然而,RNA 的不稳定性使得许多科学家对 RNA 纳米技术望而却步。阻碍 RNA 治疗学发展的其他问题包括干扰素的诱导、细胞因子的刺激和其他免疫系统的激活,以及体内药代动力学特征短暂。这篇综述将提供一些关于化学和热力学稳定性、体内半衰期和生物分布、产率和生产成本、体内毒性和副作用、特异性递药和靶向、以及内体捕获和逃逸的解决方案和展望。

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