Department of Biological Sciences, University of North Carolina at Charlotte, 9201 University City Blvd., Charlotte, NC 28223, USA.
Nanoscale Science Program, Department of Chemistry, University of North Carolina at Charlotte, Charlotte, NC 28223, USA.
Molecules. 2022 Dec 27;28(1):228. doi: 10.3390/molecules28010228.
Nucleic acid nanoparticles (NANPs) are an exciting and innovative technology in the context of both basic and biomedical research. Made of DNA, RNA, or their chemical analogs, NANPs are programmed for carrying out specific functions within human cells. NANPs are at the forefront of preventing, detecting, and treating disease. Their nucleic acid composition lends them biocompatibility that provides their cargo with enhanced opportunity for coordinated delivery. Of course, the NANP system of targeting specific cells and tissues is not without its disadvantages. Accumulation of NANPs outside of the target tissue and the potential for off-target effects of NANP-mediated cargo delivery present challenges to research and medical professionals and these challenges must be effectively addressed to provide safe treatment to patients. Importantly, development of NANPs with regulated biological activities and immunorecognition becomes a promising route for developing versatile nucleic acid therapeutics. In a basic research context, NANPs can assist investigators in fine-tuning the structure-function relationship of final formulations and in this review, we explore the practical applications of NANPs in laboratory and clinical settings and discuss how we can use established nucleic acid research techniques to design effective NANPs.
核酸纳米颗粒（NANPs）是基础和生物医学研究领域中令人兴奋且创新的技术。NANPs 由 DNA、RNA 或其化学类似物制成，经过编程可在人体细胞内执行特定功能。NANPs 处于预防、检测和治疗疾病的最前沿。其核酸组成使它们具有生物相容性，为其携带物提供了增强的协调递送机会。当然，针对特定细胞和组织的 NANP 靶向系统并非没有缺点。NANP 在靶组织外的积累以及 NANP 介导的携带物递送的潜在脱靶效应给研究和医疗专业人员带来了挑战，必须有效解决这些挑战，为患者提供安全的治疗。重要的是，具有调节生物活性和免疫识别的 NANP 的开发成为开发多功能核酸治疗的有前途的途径。在基础研究方面，NANPs 可以帮助研究人员微调最终制剂的结构-功能关系，在本综述中，我们探讨了 NANPs 在实验室和临床环境中的实际应用，并讨论了如何利用现有的核酸研究技术来设计有效的 NANPs。
