RNA 疗法的发现平台。

IF 6.8 2区 医学 Q1 PHARMACOLOGY & PHARMACY
Giulio Ciucci, Luca Braga, Serena Zacchigna
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引用次数: 0

摘要

RNA 疗法正在成为治疗目前 "无法治疗 "的分子和疾病的一个独特机会。与传统的小分子药物相比,RNA 疗法的优势、治疗意义以及有效体内给药的工具已被广泛报道,但迄今为止,人们很少关注用于发现 RNA 疗法的技术平台。在此,我们将概述用于发现 RNA 的现有平台和体内外检测方法、其优缺点及其主要应用领域,并特别关注已进入第三阶段或获得市场批准的 RNA 疗法。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Discovery platforms for RNA therapeutics.

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.

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来源期刊
CiteScore
15.40
自引率
12.30%
发文量
270
审稿时长
2.0 months
期刊介绍: The British Journal of Pharmacology (BJP) is a biomedical science journal offering comprehensive international coverage of experimental and translational pharmacology. It publishes original research, authoritative reviews, mini reviews, systematic reviews, meta-analyses, databases, letters to the Editor, and commentaries. Review articles, databases, systematic reviews, and meta-analyses are typically commissioned, but unsolicited contributions are also considered, either as standalone papers or part of themed issues. In addition to basic science research, BJP features translational pharmacology research, including proof-of-concept and early mechanistic studies in humans. While it generally does not publish first-in-man phase I studies or phase IIb, III, or IV studies, exceptions may be made under certain circumstances, particularly if results are combined with preclinical studies.
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