Williams Gregory P, Freuchet Antoine, Michaelis Tanner, Frazier April, Tran Ngan K, Rodrigues Lima-Junior João, Phillips Elizabeth J, Mallal Simon A, Litvan Irene, Goldman Jennifer G, Alcalay Roy N, Sidney John, Sulzer David, Sette Alessandro, Lindestam Arlehamn Cecilia S
Center for Autoimmunity and Inflammation, La Jolla Institute for Immunology, La Jolla, California, USA.
Aligning Science Across Parkinson's (ASAP) Collaborative Research Network, Chevy Chase, Maryland, USA.
J Clin Invest. 2024 Dec 17;135(4):e180478. doi: 10.1172/JCI180478.
Parkinson's disease (PD) is the second most prevalent neurodegenerative disorder. While there is no curative treatment, the immune system's involvement with autoimmune T cells that recognize the protein α-synuclein (α-syn) in a subset of individuals suggests new areas for therapeutic strategies. As not all patients with PD have T cells specific for α-syn, we explored additional autoantigenic targets of T cells in PD. We generated 15-mer peptides spanning several PD-related proteins implicated in PD pathology, including glucosylceramidase β 1 (GBA), superoxide dismutase 1 (SOD1), PTEN induced kinase 1 (PINK1), Parkin RBR E3 ubiquitin protein ligase (parkin), oxoglutarate dehydrogenase (OGDH), and leucine rich repeat kinase 2 (LRRK2). Cytokine production (IFN-γ, IL-5, IL-10) against these proteins was measured using a fluorospot assay and PBMCs from patients with PD and age-matched healthy controls. We identified PINK1, a regulator of mitochondrial stability, as an autoantigen targeted by T cells, as well as its unique epitopes, and their HLA restriction. The PINK1-specific T cell reactivity revealed sex-based differences, as it was predominantly found in male patients with PD, which may contribute to the heterogeneity of PD. Identifying and characterizing PINK1 and other autoinflammatory targets may lead to antigen-specific diagnostics, progression markers, and/or novel therapeutic strategies for PD.
帕金森病(PD)是第二常见的神经退行性疾病。虽然尚无治愈性治疗方法,但在一部分个体中,免疫系统与识别蛋白质α-突触核蛋白(α-syn)的自身免疫性T细胞的关联为治疗策略提供了新的方向。由于并非所有帕金森病患者都有针对α-syn的T细胞,我们探索了帕金森病中T细胞的其他自身抗原靶点。我们生成了跨越几种与帕金森病病理相关的蛋白质的15肽,包括葡糖脑苷脂酶β1(GBA)、超氧化物歧化酶1(SOD1)、PTEN诱导激酶1(PINK1)、帕金RBR E3泛素蛋白连接酶(帕金)、酮戊二酸脱氢酶(OGDH)和富含亮氨酸重复激酶2(LRRK2)。使用荧光斑点测定法和来自帕金森病患者及年龄匹配的健康对照的外周血单核细胞(PBMC),检测针对这些蛋白质的细胞因子产生(IFN-γ、IL-5、IL-10)。我们确定线粒体稳定性调节因子PINK1是T细胞靶向的自身抗原,以及其独特的表位及其HLA限制。PINK1特异性T细胞反应性显示出基于性别的差异,因为它主要在男性帕金森病患者中发现,这可能导致帕金森病的异质性。识别和表征PINK1及其他自身炎症靶点可能会带来针对帕金森病的抗原特异性诊断、疾病进展标志物和/或新型治疗策略。