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用小分子药物靶向RNA:治疗前景与化学挑战

Targeting RNA with small-molecule drugs: therapeutic promise and chemical challenges.

作者信息

Gallego J, Varani G

机构信息

MRC Laboratory of Molecular Biology, Hills Road, Cambridge CB2 2QH, UK.

出版信息

Acc Chem Res. 2001 Oct;34(10):836-43. doi: 10.1021/ar000118k.

DOI:10.1021/ar000118k
PMID:11601968
Abstract

Researchers' increasing awareness of the essential role played by RNA in many biological processes and in the progression of disease makes the discovery of new RNA targets an emerging field in drug discovery. Since most existing pharmacologically active compounds bind proteins, RNA provides nearly untapped opportunities for pharmacological development. The elucidation of the structure of the ribosome and other cellular and viral RNA motifs creates the opportunity for discovering new drug-like compounds that inhibit RNA function. However, further advances in understanding the chemistry and structure of RNA recognition are needed before these promises are fulfilled.

摘要

研究人员越来越意识到RNA在许多生物过程和疾病进展中所起的关键作用,这使得发现新的RNA靶点成为药物研发中一个新兴的领域。由于大多数现有的药理活性化合物与蛋白质结合,RNA为药物研发提供了几乎尚未开发的机会。核糖体以及其他细胞和病毒RNA基序结构的阐明为发现抑制RNA功能的新型类药物化合物创造了机会。然而,在实现这些前景之前,还需要在理解RNA识别的化学和结构方面取得进一步进展。

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Targeting RNA with small-molecule drugs: therapeutic promise and chemical challenges.用小分子药物靶向RNA:治疗前景与化学挑战
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RNA as a drug target.RNA作为药物靶点。
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The bacterial ribosome, a promising focus for structure-based drug design.细菌核糖体是基于结构的药物设计的一个有前景的研究重点。
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