Guangdong Provincial Key Laboratory of Malignant Tumor Epigenetics and Gene Regulation, Guangdong-Hong Kong Joint Laboratory for RNA Medicine, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou 510120, China; Nanhai Clinical Translational Center, Sun Yat-sen Memorial Hospital, Foshan 528200, China.
Guangdong Provincial Key Laboratory of Malignant Tumor Epigenetics and Gene Regulation, Guangdong-Hong Kong Joint Laboratory for RNA Medicine, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou 510120, China; Nanhai Clinical Translational Center, Sun Yat-sen Memorial Hospital, Foshan 528200, China; Breast Tumor Center, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou 510120, China.
Cell Rep Med. 2024 May 21;5(5):101555. doi: 10.1016/j.xcrm.2024.101555. Epub 2024 May 13.
RNA molecules have emerged as promising clinical therapeutics due to their ability to target "undruggable" proteins or molecules with high precision and minimal side effects. Nevertheless, the primary challenge in RNA therapeutics lies in rapid degradation and clearance from systemic circulation, the inability to traverse cell membranes, and the efficient intracellular delivery of bioactive RNA molecules. In this review, we explore the implications of RNAs in diseases and provide a chronological overview of the development of RNA therapeutics. Additionally, we summarize the technological advances in RNA-screening design, encompassing various RNA databases and design platforms. The paper then presents an update on FDA-approved RNA therapeutics and those currently undergoing clinical trials for various diseases, with a specific emphasis on RNA medicine and RNA vaccines.
RNA 分子因其能够以高精度靶向“不可成药”的蛋白质或分子,且副作用极小,已成为有前途的临床治疗药物。然而,RNA 治疗学的主要挑战在于其在体内循环中迅速降解和清除、无法穿透细胞膜以及生物活性 RNA 分子的有效细胞内递呈。在这篇综述中,我们探讨了 RNA 在疾病中的作用,并按时间顺序概述了 RNA 治疗学的发展。此外,我们总结了 RNA 筛选设计的技术进展,包括各种 RNA 数据库和设计平台。本文随后介绍了已获得 FDA 批准的 RNA 治疗药物,以及目前正在针对各种疾病进行临床试验的药物,特别强调了 RNA 药物和 RNA 疫苗。