Ribonucleic acid (RNA) therapeutics are being actively researched as a therapeutic modality in preclinical and clinical studies. They have become one of the most ubiquitously known and discussed therapeutics in recent years in part due to the ongoing coronavirus pandemic. Since the first approval in 1998, research on RNA therapeutics has progressed to discovering new therapeutic targets and delivery strategies to enhance their safety and efficacy. Here, we provide an overview of the current clinically relevant RNA therapeutics, mechanistic basis of their function, and strategies to improve their clinical use. We discuss the 17 approved RNA therapeutics and perform an in-depth analysis of the 222 ongoing clinical trials, with an emphasis on their respective mechanisms and disease areas. We also provide perspectives on the challenges for clinical translation of RNA therapeutics and suggest potential strategies to address these challenges.

中文翻译：

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RNA疗法在临床中的应用


核糖核酸 (RNA) 疗法作为临床前和临床研究的治疗方式正在积极研究。近年来，它们已成为最广为人知和讨论最多的疗法之一，部分原因是持续的冠状病毒大流行。自 1998 年首次获得批准以来，RNA 疗法的研究已取得进展，发现了新的治疗靶点和递送策略，以提高其安全性和有效性。在这里，我们概述了当前临床相关的 RNA 疗法、其功能的机制基础以及改善其临床应用的策略。我们讨论了 17 种已批准的 RNA 疗法，并对 222 项正在进行的临床试验进行了深入分析，重点是它们各自的机制和疾病领域。我们还提供了有关 RNA 疗法临床转化挑战的观点，并提出了应对这些挑战的潜在策略。