Due to a series of systemic and intracellular obstacles in nucleic acid (NA) therapy, including fast degradation in blood, renal clearance, poor cellular uptake, and inefficient endosomal escape, NAs may need delivery methods to transport to the cell nucleus or cytosol to be effective. Advanced nanoscale biotechnology-associated strategies, such as controlling the particle size, charge, drug loading, response to environmental signals, or other physical/chemical properties of delivery carriers, have provided great help for the and delivery of NA therapeutics. In this review, we introduce the characteristics of different NA modalities and illustrate how advanced nanoscale biotechnology assists NA therapy. The specific features and challenges of various nanocarriers in clinical and preclinical studies are summarized and discussed. With the help of advanced nanoscale biotechnology, some of the major barriers to the development of NA therapy will eventually be overcome in the near future.

中文翻译：

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利用先进纳米生物技术改进核酸疗法


由于核酸（NA）治疗中存在一系列全身和细胞内障碍，包括血液中的快速降解、肾清除、细胞摄取差以及内体逃逸效率低下，NAs可能需要递送方法来转运至细胞核或细胞质中有效的。先进的纳米级生物技术相关策略，例如控制递送载体的粒径、电荷、载药量、对环境信号的响应或其他物理/化学特性，为NA治疗剂的递送提供了巨大的帮助。在这篇综述中，我们介绍了不同 NA 模式的特点，并说明先进的纳米生物技术如何协助 NA 治疗。总结和讨论了各种纳米载体在临床和临床前研究中的具体特征和挑战。借助先进的纳米级生物技术，NA疗法发展的一些主要障碍最终将在不久的将来被克服。