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身体首发和脑首发突触核蛋白病中α-突触核蛋白毒株变异性

Alpha-Synuclein Strain Variability in Body-First and Brain-First Synucleinopathies.

作者信息

Just Mie Kristine, Gram Hjalte, Theologidis Vasileios, Jensen Poul Henning, Nilsson K Peter R, Lindgren Mikael, Knudsen Karoline, Borghammer Per, Van Den Berge Nathalie

机构信息

Institute for Clinical Medicine, Aarhus University, Aarhus, Denmark.

Nuclear Medicine and PET, Aarhus University Hospital, Aarhus, Denmark.

出版信息

Front Aging Neurosci. 2022 May 26;14:907293. doi: 10.3389/fnagi.2022.907293. eCollection 2022.

Abstract

Pathogenic alpha-synuclein (asyn) aggregates are a defining feature of neurodegenerative synucleinopathies, which include Parkinson's disease, Lewy body dementia, pure autonomic failure and multiple system atrophy. Early accurate differentiation between these synucleinopathies is challenging due to the highly heterogeneous clinical profile at early prodromal disease stages. Therefore, diagnosis is often made in late disease stages when a patient presents with a broad range of motor and non-motor symptoms easing the differentiation. Increasing data suggest the clinical heterogeneity seen in patients is explained by the presence of distinct asyn strains, which exhibit variable morphologies and pathological functions. Recently, asyn seed amplification assays (PMCA and RT-QuIC) and conformation-specific ligand assays have made promising progress in differentiating between synucleinopathies in prodromal and advanced disease stages. Importantly, the cellular environment is known to impact strain morphology. And, asyn aggregate pathology can propagate trans-synaptically along the brain-body axis, affecting multiple organs and propagating through multiple cell types. Here, we present our hypothesis that the changing cellular environments, an asyn seed may encounter during its brain-to-body or body-to-brain propagation, may influence the structure and thereby the function of the aggregate strains developing within the different cells. Additionally, we aim to review strain characteristics of the different synucleinopathies in clinical and preclinical studies. Future preclinical animal models of synucleinopathies should investigate if asyn strain morphology is altered during brain-to-body and body-to-brain spreading using these seeding amplification and conformation-specific assays. Such findings would greatly deepen our understanding of synucleinopathies and the potential link between strain and phenotypic variability, which may enable specific diagnosis of different synucleinopathies in the prodromal phase, creating a large therapeutic window with potential future applications in clinical trials and personalized therapeutics.

摘要

致病性α-突触核蛋白(α-syn)聚集体是神经退行性突触核蛋白病的一个决定性特征,这些疾病包括帕金森病、路易体痴呆、单纯自主神经功能衰竭和多系统萎缩。由于在疾病前驱期的临床特征高度异质性,早期准确区分这些突触核蛋白病具有挑战性。因此,诊断通常在疾病晚期进行,此时患者出现广泛的运动和非运动症状,便于区分。越来越多的数据表明,患者中观察到的临床异质性是由不同的α-syn毒株的存在所解释的,这些毒株表现出可变的形态和病理功能。最近,α-syn种子扩增检测(PMCA和RT-QuIC)以及构象特异性配体检测在区分前驱期和晚期疾病阶段的突触核蛋白病方面取得了有希望的进展。重要的是,已知细胞环境会影响毒株形态。而且,α-syn聚集体病理学可以沿脑-体轴跨突触传播,影响多个器官并通过多种细胞类型传播。在这里,我们提出我们的假设,即α-syn种子在其脑-体或体-脑传播过程中可能遇到的不断变化的细胞环境,可能会影响聚集体毒株在不同细胞内形成的结构,进而影响其功能。此外,我们旨在回顾临床和临床前研究中不同突触核蛋白病的毒株特征。未来突触核蛋白病的临床前动物模型应该使用这些种子扩增和构象特异性检测来研究α-syn毒株形态在脑-体和体-脑传播过程中是否发生改变。这些发现将极大地加深我们对突触核蛋白病的理解以及毒株与表型变异性之间的潜在联系,这可能使我们能够在疾病前驱期对不同的突触核蛋白病进行特异性诊断,从而创造一个巨大的治疗窗口,在未来的临床试验和个性化治疗中具有潜在应用价值。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3182/9178288/8f9a27563633/fnagi-14-907293-g0001.jpg

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