Systematically Studying the Effect of Small Molecules Interacting with RNA in Cellular and Preclinical Models.
Systematically Studying the Effect of Small Molecules Interacting with RNA in Cellular and Preclinical Models.
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DOI：
10.1021/acschembio.1c00014
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发表时间：
2021-07-16
影响因子：
4
通讯作者：
Disney MD
中科院分区：
文献类型：
--
作者：
Bush JA;Williams CC;Meyer SM;Tong Y;Haniff HS;Childs-Disney JL;Disney MD
The interrogation and manipulation of biological systems by small molecules is a powerful approach in chemical biology. Ideal compounds selectively engage a target and mediate a downstream phenotypic response. Although historically small molecule drug discovery has focused on proteins and enzymes, targeting RNA is an attractive therapeutic alternative, as many disease-causing or -associated RNAs have been identified through genome-wide association studies. As the field of RNA chemical biology emerges, the systematic evaluation of target validation and modulation of target-associated pathways is of paramount importance. In this Review, through an examination of case studies, we outline the experimental characterization, including methods and tools, to evaluate comprehensively the impact of small molecules that target RNA on cellular phenotype.
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DOI：
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通讯作者：
Disney MD
DOI：
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发表时间：
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影响因子：
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通讯作者：
Disney, Matthew D.