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Updated: Jan 10, 2026

Exploring Sequence Space to Identify Binding Sites for Regulatory RNA-Binding Proteins

Exploring Sequence Space to Identify Binding Sites for Regulatory RNA-Binding Proteins

Published on: August 9, 2019

聚焦RNA的DNA编码图书馆构建,选和整合对接识别致病性r的生物活性干 (G₄C₂) ^expRNA

Xueyi Yang^1,2, Amirhossein Taghavi¹, Yoshihiro Akahori¹

¹Department of Chemistry, The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology, 130 Scripps Way, Jupiter, FL 33458, United States.

bioRxiv : the preprint server for biology

|November 24, 2025

概括

研究人员开发了一个DNA编码库 (DEL),以发现针对有毒RNA重复的小分子. 这种方法成功地识别和优化了用于诸如肌缩侧面硬化症 (ALS) 和前性痴呆症 (FTD) 等疾病的药物.

科学领域:

药用化学医学化学
在RNA治疗方面,RNA疗法.
药物发现药物发现药物发现

背景情况:

与疾病相关的RNA正在成为小分子药物的关键治疗标.
DNA编码图书馆 (DELs) 对识别RNA结合小分子具有前途意义,补充了它们在蛋白质连接体发现中的既定用途.

研究的目的:

为了构建一个多样化的,固相DEL,富含RNA结合支架.
为了识别与ALS和FTD相关的有毒r(G4C2) RNA重复扩张的小分子配体.
展示DEL技术与用于合理RNA向药物设计的计算方法的集成.

主要方法:

合成了一种专门的DEL,并对r(G4C2) RNA进行选.
使用大规模的分子对接,物理化学分析和结构-活性关系 (SAR) 研究来分析DEL选择结果.
化合物优化以对接姿势和NMR结构为指导.

主要成果:

在分子对接预测和实验丰富数据之间发现了显著的相关性.
确定的化合物被优化为具有更好的结合亲和力和生物活性的类似物.
该研究验证了用于RNA配体发现的联合DEL和计算方法.

结论:

开发的平台有效地识别了向RNA的小分子.
这种综合方法加速了RNA向治疗的合理设计和优化.
这些发现突出了发现RNA介导疾病新型治疗方法的多功能策略.

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PAR-CliP - A Method to Identify Transcriptome-wide the Binding Sites of RNA Binding Proteins

PAR-CliP - A Method to Identify Transcriptome-wide the Binding Sites of RNA Binding Proteins

Published on: July 2, 2010

Novel RNA-Binding Proteins Isolation by the RaPID Methodology

Novel RNA-Binding Proteins Isolation by the RaPID Methodology

Published on: September 30, 2016

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