The beginning of a new era:pioneering direct screens for RNA modulators
作者机构：Institute of Biochemistry IIGoethe University FrankfurtFaculty of MedicineFrankfurt am MainGermany Frankfurt Cancer InstituteFrankfurt am MainGermany CardioPulmonary InstituteFrankfurt am MainGermany
出 版 物：《Signal Transduction and Targeted Therapy》 (信号转导与靶向治疗（英文）)
年 卷 期：2022年第7卷第8期
页 面：2660-2661页
核心收录：
学科分类：1007[医学-药学(可授医学、理学学位)] 1006[医学-中西医结合] 100706[医学-药理学] 100602[医学-中西医结合临床] 10[医学]
基 金：supported by grants from the European Research Council(ERC StG 803565) the Deutsche Forschungsgemeinschaft(DFG,German Research Foundation)Project-ID 390339347(Emmy Noether Programme) the EnABLE consortium funded by the Hessian Ministry for Arts and Sciences
摘 要：In a recent study published in Nature,1 Aguilar et *** the small molecule X1 that targets the long non-coding RNA *** X1 binding,Xist changes its structure,which prevents its role in X-chromosome inactivation(XCI).The vast majority of small molecule screens in drug discovery focus on targeting proteins.2,3 However,protein coding sequences merely constitute~1.5%of the human genome and only a small fraction of these proteins are currently targetable.2 In contrast,90%of the human genome is transcribed into RNA in different forms,which makes RNAs compelling targets for small molecule drugs.3 Modulating mRNAs with small molecules could be an alternative route to change the levels of the corresponding protein products and their activities,especially for currently undruggable *** the diverse functions carried out by different classes of RNAs in cells,altering RNAs would provide a broad range of potential drug targets in the human *** modulating RNAs with small molecules carries a number of advantages,identifying RNA-targeting small molecules has proven difficult;previous compounds had been identified indirectly by phenotypic screens.1–3 Systematic approaches to screen for RNAtargeting compounds had been lacking.
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