Ciucci Giulio, Braga Luca, Zacchigna Serena
Cardiovascular Biology Laboratory, International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
Functional Cell Biology Laboratory, International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
Br J Pharmacol. 2025 Jan;182(2):281-295. doi: 10.1111/bph.16424. Epub 2024 May 17.
RNA therapeutics are emerging as a unique opportunity to drug currently "undruggable" molecules and diseases. While their advantages over conventional, small molecule drugs, their therapeutic implications and the tools for their effective in vivo delivery have been extensively reviewed, little attention has been so far paid to the technological platforms exploited for the discovery of RNA therapeutics. Here, we provide an overview of the existing platforms and ex vivo assays for RNA discovery, their advantages and disadvantages, as well as their main fields of application, with specific focus on RNA therapies that have reached either phase 3 or market approval. LINKED ARTICLES: This article is part of a themed issue Non-coding RNA Therapeutics. To view the other articles in this section visit http://onlinelibrary.wiley.com/doi/10.1111/bph.v182.2/issuetoc.
RNA疗法正成为针对目前“难以成药”的分子和疾病开发药物的独特契机。尽管相较于传统小分子药物，RNA疗法的优势、其治疗意义以及体内有效递送工具已得到广泛综述，但迄今为止，人们对用于发现RNA疗法的技术平台关注甚少。在此，我们概述了用于RNA发现的现有平台和体外测定方法、它们的优缺点及其主要应用领域，并特别关注已进入3期临床试验或已获市场批准的RNA疗法。相关文章：本文是主题为“非编码RNA疗法”的特刊的一部分。若要查看本部分的其他文章，请访问http://onlinelibrary.wiley.com/doi/10.1111/bph.v182.2/issuetoc 。
