Martin Schuler, Grischa Gerwert, Marvin Mann, Nathalie Woitzik, Lennart Langenhoff, Diana Hubert, Deniz Duman, Adrian Höveler, Sandy Galkowski, Jonas Simon, Robin Denz, Sandrina Weber, Eun-Hae Kwon, Robin Wanka, Carsten Kötting, Jörn Güldenhaupt, Léon Beyer, Lars Tönges, Brit Mollenhauer, Klaus Gerwert
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引用次数: 0
摘要
α -突触核蛋白(αSyn)的错误折叠和聚集在帕金森病(PD)的病理生理中起着关键作用。尽管在诊断方面取得了相当大的进步,但帕金森病的早期和鉴别诊断仍然是一个重大挑战。我们创新了免疫红外传感器(iRS)平台来测量αSyn错误折叠。我们分析了来自PD病例、非典型帕金森病和疾病对照的两个队列的脑脊液(CSF)。通过红外光谱测定αSyn错误折叠,我们获得了PD/MSA与对照组分离的AUC为0.90 (n = 134, 95% CI 0.85-0.96)。使用两个阈值将个体划分为不受错误折叠影响/不受错误折叠影响的个体,并在两者之间设置一个中间区域。比较受影响/未受影响的病例,对照与PD/MSA病例的分类灵敏度为97%,特异性为92%。光谱数据显示,PD和MSA患者的α-螺旋/随机线圈αSyn折叠错误,从对照组的α-螺旋/随机线圈αSyn折叠到β-薄片富集αSyn。此外,第一亚组分析暗示在临床重叠病例中患者分层的可能性。iRS直接测量所有αSyn构象,是αSyn种子扩增法(SAAs)的补充,然而,SAAs只能扩增具有播种能力的构象。
Alpha-synuclein misfolding as fluid biomarker for Parkinson's disease measured with the iRS platform.
Misfolding and aggregation of alpha-synuclein (αSyn) play a key role in the pathophysiology of Parkinson's disease (PD). Despite considerable advances in diagnostics, an early and differential diagnosis of PD still represents a major challenge. We innovated the immuno-infrared sensor (iRS) platform for measuring αSyn misfolding. We analyzed cerebrospinal fluid (CSF) from two cohorts comprising PD cases, atypical Parkinsonian disorders, and disease controls. We obtained an AUC of 0.90 (n = 134, 95% CI 0.85-0.96) for separating PD/MSA from controls by determination of the αSyn misfolding by iRS. Using two thresholds divided individuals as unaffected/affected by misfolding with an intermediate area in between. Comparing the affected/unaffected cases, controls versus PD/MSA cases were classified with 97% sensitivity and 92% specificity. The spectral data revealed misfolding from an α-helical/random-coil αSyn in controls to β-sheet enriched αSyn in PD and MSA cases. Moreover, a first subgroup analysis implied the potential for patient stratification in clinically overlapping cases. The iRS, directly measuring all αSyn conformers, is complementary to the αSyn seed-amplification assays (SAAs), which however only amplify seeding competent conformers.
期刊介绍:
EMBO Molecular Medicine is an open access journal in the field of experimental medicine, dedicated to science at the interface between clinical research and basic life sciences. In addition to human data, we welcome original studies performed in cells and/or animals provided they demonstrate human disease relevance.
To enhance and better specify our commitment to precision medicine, we have expanded the scope of EMM and call for contributions in the following fields:
Environmental health and medicine, in particular studies in the field of environmental medicine in its functional and mechanistic aspects (exposome studies, toxicology, biomarkers, modeling, and intervention).
Clinical studies and case reports - Human clinical studies providing decisive clues how to control a given disease (epidemiological, pathophysiological, therapeutic, and vaccine studies). Case reports supporting hypothesis-driven research on the disease.
Biomedical technologies - Studies that present innovative materials, tools, devices, and technologies with direct translational potential and applicability (imaging technologies, drug delivery systems, tissue engineering, and AI)