Department of Molecular Medicine, Faculty of Pharmacy and Medicine, Sapienza University of Rome, Viale Regina Elena 324, 00161 Rome, Italy.
National Institute for Infectious Diseases "L. Spallanzani", Via Portuense 292, 00149 Rome, Italy.
Int J Mol Sci. 2022 Jul 5;23(13):7471. doi: 10.3390/ijms23137471.
The growing knowledge on several classes of non-coding RNAs (ncRNAs) and their different functional roles has aroused great interest in the scientific community. Beyond the Central Dogma of Biology, it is clearly known that not all RNAs code for protein products, and they exert a broader repertoire of biological functions. As described in this review, ncRNAs participate in gene expression regulation both at transcriptional and post-transcriptional levels and represent critical elements driving and controlling pathophysiological processes in multicellular organisms. For this reason, in recent years, a great boost was given to ncRNA-based strategies with potential therapeutic abilities, and nowadays, the use of RNA molecules is experimentally validated and actually exploited in clinics to counteract several diseases. In this review, we summarize the principal classes of therapeutic ncRNA molecules that are potentially implied in disease onset and progression, which are already used in clinics or under clinical trials, highlighting the advantages and the need for a targeted therapeutic strategy design. Furthermore, we discuss the benefits and the limits of RNA therapeutics and the ongoing development of delivery strategies to limit the off-target effects and to increase the translational application.
越来越多的关于几类非编码 RNA（ncRNAs）及其不同功能作用的知识引起了科学界的极大兴趣。除了生物学的中心法则之外，显然并非所有的 RNA 都编码蛋白质产物，它们发挥着更广泛的生物学功能。正如本综述所述，ncRNAs 参与转录和转录后水平的基因表达调控，是驱动和控制多细胞生物生理病理过程的关键因素。出于这个原因，近年来，基于 ncRNA 的具有潜在治疗能力的策略得到了极大的推动，如今，RNA 分子的应用在实验上得到了验证，并在临床上实际用于对抗多种疾病。在本综述中，我们总结了可能涉及疾病发生和进展的主要治疗性 ncRNA 分子类别，这些分子已经在临床上使用或正在临床试验中，强调了靶向治疗策略设计的优势和必要性。此外，我们还讨论了 RNA 治疗的益处和局限性，以及正在开发的输送策略，以限制脱靶效应并增加转化应用。
