Neuroregeneration and Stem Cell Programs, Institute for Cell Engineering, Johns Hopkins University School of Medicine, Baltimore, MD, 21205, USA.
Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, 21205, USA.
Nat Commun. 2024 May 31;15(1):4663. doi: 10.1038/s41467-024-49016-3.
Pathologic α-synuclein (α-syn) spreads from cell-to-cell, in part, through binding to the lymphocyte-activation gene 3 (Lag3). Here we report that amyloid β precursor-like protein 1 (Aplp1) interacts with Lag3 that facilitates the binding, internalization, transmission, and toxicity of pathologic α-syn. Deletion of both Aplp1 and Lag3 eliminates the loss of dopaminergic neurons and the accompanying behavioral deficits induced by α-syn preformed fibrils (PFF). Anti-Lag3 prevents the internalization of α-syn PFF by disrupting the interaction of Aplp1 and Lag3, and blocks the neurodegeneration induced by α-syn PFF in vivo. The identification of Aplp1 and the interplay with Lag3 for α-syn PFF induced pathology deepens our insight about molecular mechanisms of cell-to-cell transmission of pathologic α-syn and provides additional targets for therapeutic strategies aimed at preventing neurodegeneration in Parkinson's disease and related α-synucleinopathies.
病理性 α-突触核蛋白(α-syn)通过与淋巴细胞激活基因 3(Lag3)结合,在细胞间传播,部分是通过这种方式传播的。在这里,我们报告淀粉样前体样蛋白 1(Aplp1)与 Lag3 相互作用,促进了病理性 α-syn 的结合、内化、传递和毒性。Aplp1 和 Lag3 的缺失消除了由 α-syn 预形成纤维(PFF)引起的多巴胺能神经元丧失和伴随的行为缺陷。抗 Lag3 通过破坏 Aplp1 和 Lag3 的相互作用来阻止 α-syn PFF 的内化,并阻止体内由 α-syn PFF 引起的神经退行性变。Aplp1 的鉴定及其与 Lag3 的相互作用对于 α-syn PFF 诱导的病理学加深了我们对病理性 α-syn 细胞间传播的分子机制的认识,并为旨在预防帕金森病和相关 α-突触核蛋白病中神经退行性变的治疗策略提供了额外的靶点。