School of Biological Sciences and Engineering, Yachay Tech University, 100119, San Miguel de Urcuquí, Ecuador.
GenScript, Jiangsu Province, 212000, People's Republic of China.
J Ind Microbiol Biotechnol. 2021 Dec 23;48(9-10). doi: 10.1093/jimb/kuab063.
Although the study of ribonucleic acid (RNA) therapeutics started decades ago, for many years, this field of research was overshadowed by the growing interest in DNA-based therapies. Nowadays, the role of several types of RNA in cell regulation processes and the development of various diseases have been elucidated, and research in RNA therapeutics is back with force. This short literature review aims to present general aspects of many of the molecules currently used in RNA therapeutics, including in vitro transcribed mRNA (IVT mRNA), antisense oligonucleotides (ASOs), aptamers, small interfering RNAs (siRNAs), and microRNAs (miRNAs). In addition, we describe the state of the art of technologies applied for synthetic RNA manufacture and delivery. Likewise, we detail the RNA-based therapies approved by the FDA so far, as well as the ongoing clinical investigations. As a final point, we highlight the current and potential advantages of working on RNA-based therapeutics and how these could lead to a new era of accessible and personalized healthcare.
尽管 RNA 疗法的研究已经开展了几十年，但在很长一段时间里，这个研究领域都被人们对基于 DNA 的疗法的日益浓厚的兴趣所掩盖。如今，人们已经阐明了几种类型的 RNA 在细胞调控过程和各种疾病发展中的作用，RNA 疗法的研究也重新兴起。这篇简短的文献综述旨在介绍当前用于 RNA 疗法的许多分子的一般方面，包括体外转录的 mRNA（IVT mRNA）、反义寡核苷酸（ASO）、适体、小干扰 RNA（siRNA）和 microRNA（miRNA）。此外，我们还描述了用于合成 RNA 制造和递送的技术的最新进展。同样，我们详细介绍了迄今为止 FDA 批准的基于 RNA 的疗法，以及正在进行的临床研究。最后，我们强调了目前基于 RNA 的疗法的优势和潜力，以及这些优势如何引领一个新的、普及的和个性化的医疗保健时代。
