Carroll Emma C, Yang Hyunjun, Powell Wyatt C, Charvat Annemarie F, Oehler Abby, Jones Julia G, Montgomery Kelly M, Yung Anthony, Millbern Zoe, Taylor Alexander I P, Wilkinson Martin, Ranson Neil A, Radford Sheena E, Vinueza Nelson R, DeGrado William F, Mordes Daniel A, Condello Carlo, Gestwicki Jason E
Department of Chemistry, San José State University, San José, CA, USA.
Institute for Neurodegenerative Diseases, University of California, San Francisco, San Francisco, CA, USA.
Nat Chem. 2025 Aug 14. doi: 10.1038/s41557-025-01889-7.
Aggregation of microtubule-associated protein tau into conformationally distinct fibrils underpins neurodegenerative tauopathies. Fluorescent probes (fluoroprobes) such as thioflavin T have been essential tools for studying tau aggregation; however, most of them do not discriminate between amyloid fibril conformations (polymorphs). This gap is due, in part, to a lack of high-throughput methods for screening large, diverse chemical collections. Here we leverage advances in protein-adaptive differential scanning fluorimetry to screen the Aurora collection of 300+ fluoroprobes against multiple synthetic fibril polymorphs, including those formed from tau, α-synuclein and islet amyloid polypeptide. This screen-coupled with excitation-multiplexed bright-emission recording (EMBER) imaging and orthogonal secondary assays-revealed pan-fibril-binding chemotypes, as well as fluoroprobes selective for fibril subsets. One fluoroprobe recognized tau pathology in ex vivo brain slices from Alzheimer's disease and rodent models. We propose that these scaffolds represent entry points for developing fibril-selective ligands.
微管相关蛋白tau聚集成构象不同的纤维是神经退行性tau蛋白病的基础。诸如硫黄素T之类的荧光探针一直是研究tau聚集的重要工具;然而,它们中的大多数无法区分淀粉样纤维的构象(多晶型物)。这种差距部分归因于缺乏用于筛选大量、多样化学物质库的高通量方法。在这里,我们利用蛋白质适应性差示扫描荧光法的进展,针对多种合成纤维多晶型物筛选300多种荧光探针的Aurora文库,这些多晶型物包括由tau、α-突触核蛋白和胰岛淀粉样多肽形成的纤维。该筛选与激发多重明亮发射记录(EMBER)成像及正交二级分析相结合,揭示了泛纤维结合化学类型以及对纤维亚群具有选择性的荧光探针。一种荧光探针在阿尔茨海默病和啮齿动物模型的离体脑切片中识别出tau病理学特征。我们认为,这些支架代表了开发纤维选择性配体的切入点。