Liu Yuxuan, Ou Yaohui, Hou Linlin
School of Medicine, Shenzhen Campus of Sun Yat-sen University, Shenzhen 518107, China.
Curr Issues Mol Biol. 2024 Dec 31;47(1):22. doi: 10.3390/cimb47010022.
Nucleic acids, as carriers of genetic information, have found wide applications in both medical and research fields, including gene editing, disease diagnostics, and drug development. Among various types of nucleic acids, RNA offers greater versatility compared to DNA due to its single-stranded structure, ability to directly encode proteins, and high modifiability for targeted therapeutic and regulatory applications. Despite its promising potential in biomedicine, RNA-based medicine still faces several challenges. Notably, one of the most significant technical hurdles is achieving efficient and targeted RNA delivery while minimizing immune responses. Various strategies have been developed for RNA delivery, including viral vectors, virus-like particles (VLPs), lipid nanoparticles (LNPs), and extracellular vesicles (EVs). In this review, we explore the applications of these delivery methods, highlight their advantages and limitations, and discuss recent research advancements, providing insights for the future of RNA-based therapeutics.
核酸作为遗传信息的载体，在医学和研究领域都有广泛应用，包括基因编辑、疾病诊断和药物开发。在各类核酸中，RNA因其单链结构、直接编码蛋白质的能力以及在靶向治疗和调控应用中的高可修饰性，相比DNA具有更大的通用性。尽管RNA在生物医学领域具有广阔前景，但基于RNA的药物仍面临一些挑战。值得注意的是，最重大的技术障碍之一是在尽量减少免疫反应的同时实现高效且靶向的RNA递送。已开发出多种RNA递送策略，包括病毒载体、病毒样颗粒（VLP）、脂质纳米颗粒（LNP）和细胞外囊泡（EV）。在本综述中，我们探讨这些递送方法的应用，突出它们的优点和局限性，并讨论近期的研究进展，为基于RNA的治疗的未来发展提供见解。
