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.

中文翻译：

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RNA疗法的无限可能

尽管核糖核酸 (RNA) 疗法的研究始于几十年前，但多年来，这一研究领域因对基于 DNA 的疗法日益增长的兴趣而黯然失色。如今，几种类型的RNA在细胞调控过程中的作用和各种疾病的发展已被阐明，RNA治疗学的研究又回来了。这篇简短的文献综述旨在介绍目前用于 RNA 治疗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 制造和递送的最新技术。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。最后一点，我们强调了基于 RNA 的疗法的当前和潜在优势，以及这些优势如何引领可访问和个性化医疗保健的新时代。