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首页> 外文期刊>Journal of the American Chemical Society >Targeting RNA with Small Molecules To Capture Opportunities at the Intersection of Chemistry, Biology, and Medicine
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Targeting RNA with Small Molecules To Capture Opportunities at the Intersection of Chemistry, Biology, and Medicine

机译：用小分子靶向RNA，以捕获化学，生物学和医学交叉点的机会

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摘要

The biology of healthy and disease-affected cells is often mediated by RNA structures, desirable targets for small molecule chemical probes and lead medicines. Although structured regions are found throughout the transcriptome, some even with demonstrated functionality, human RNAs are considered recalcitrant to small molecule targeting. However, targeting structured regions with small molecules provides an important alternative to oligonucleotides that target sequence. In this Perspective, we describe challenges and progress in developing small molecules interacting with RNA (SMIRNAs) to capture their significant opportunities at the intersection of chemistry, biology, and medicine. Key to establishing a new paradigm in chemical biology and medicine is the development of methods to obtain, preferably by design, bioactive compounds that modulate RNA targets and companion methods that validate their direct effects in cells and pre-clinical models. While difficult, demonstration of direct target engagement in the complex cellular milieu, along with methods to establish modes of action, is required to push this field forward. We also describe frameworks for accelerated advancements in this burgeoning area, their implications, key new technologies for development of SMIRNAs, and milestones that have led to broader acceptance of RNA as a small molecule druggable target.
机译：健康和疾病影响的细胞的生物学通常由RNA结构介导，小分子化学探针和铅药物的理想靶标。尽管在整个转录组中发现了结构区域，但是一些甚至具有证明的功能，但人RNA被认为是顽固的小分子靶向。然而，靶向具有小分子的结构化区域为靶向序列的寡核苷酸提供了重要的替代方案。在这种观点中，我们描述了开发与RNA（Smirnas）相互作用的小分子进行挑战和进展，以捕获化学，生物学和医学交叉口的重要机会。在化学生物学和药物中建立新的范例的关键是开发方法，优选地通过设计，调节验证其在细胞和临床前模型中的直接效应的RNA靶标和伴侣方法的方法。虽然困难，在复杂的蜂窝环境中直接目标接触的演示以及建立行动模式的方法，需要推动该场。我们还描述了在这一兴奋地区的加速进步的框架，其影响，开发斯波纳斯的关键新技术，以及导致更广泛接受RNA作为小分子可用性靶标的里程碑的新技术。

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  • 来源
    《Journal of the American Chemical Society》 |2019年第17期|6776-6790|共15页
  • 作者

    Disney Matthew D.;

  • 作者单位

    Scripps Res Inst Dept Chem Jupiter FL 33458 USA;

  • 收录信息 美国《科学引文索引》(SCI);美国《工程索引》(EI);美国《生物学医学文摘》(MEDLINE);美国《化学文摘》(CA);
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  • 正文语种 eng
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