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化学配体与多态α-突触核蛋白淀粉样纤维的结合适应性。

Binding adaptability of chemical ligands to polymorphic α-synuclein amyloid fibrils.

机构信息

Interdisciplinary Research Center on Biology and Chemistry, Shanghai Institute of Organic Chemistry, Chinese Academy of Sciences, Shanghai 201210, China.

Bio-X Institutes, Key Laboratory for the Genetics of Developmental and Neuropsychiatric Disorders (Ministry of Education), Shanghai Jiao Tong University, Shanghai 200030, China.

出版信息

Proc Natl Acad Sci U S A. 2024 Aug 27;121(35):e2321633121. doi: 10.1073/pnas.2321633121. Epub 2024 Aug 22.

Abstract

α-synuclein (α-syn) assembles into structurally distinct fibril polymorphs seen in different synucleinopathies, such as Parkinson's disease and multiple system atrophy. Targeting these unique fibril structures using chemical ligands holds diagnostic significance for different disease subtypes. However, the molecular mechanisms governing small molecules interacting with different fibril polymorphs remain unclear. Here, we investigated the interactions of small molecules belonging to four distinct scaffolds, with different α-syn fibril polymorphs. Using cryo-electron microscopy, we determined the structures of these molecules when bound to the fibrils formed by E46K mutant α-syn and compared them to those bound with wild-type α-syn fibrils. Notably, we observed that these ligands exhibit remarkable binding adaptability, as they engage distinct binding sites across different fibril polymorphs. While the molecular scaffold primarily steered the binding locations and geometries on specific sites, the conjugated functional groups further refined this adaptable binding by fine-tuning the geometries and binding sites. Overall, our finding elucidates the adaptability of small molecules binding to different fibril structures, which sheds light on the diagnostic tracer and drug developments tailored to specific pathological fibril polymorphs.

摘要

α-突触核蛋白(α-syn)会组装成不同突触核病(如帕金森病和多系统萎缩)中可见的结构不同的纤维多态体。使用化学配体靶向这些独特的纤维结构对于不同的疾病亚型具有诊断意义。然而,控制小分子与不同纤维多态体相互作用的分子机制仍不清楚。在这里,我们研究了属于四个不同支架的小分子与不同的α-syn 纤维多态体的相互作用。使用冷冻电子显微镜,我们确定了这些小分子与 E46K 突变α-syn 形成的纤维结合时的结构,并将其与与野生型α-syn 纤维结合时的结构进行了比较。值得注意的是,我们观察到这些配体表现出显著的结合适应性,因为它们在不同的纤维多态体上结合到不同的结合位点。虽然分子支架主要控制特定部位的结合位置和几何形状,但共轭的功能基团通过微调几何形状和结合位点进一步细化了这种适应性结合。总的来说,我们的发现阐明了小分子与不同纤维结构结合的适应性,这为针对特定病理纤维多态体的诊断示踪剂和药物开发提供了启示。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0eaf/11363296/c09f4bb92d17/pnas.2321633121fig01.jpg

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