Kuo Grace, Kumbhar Ramhari, Blair William, Dawson Valina L, Dawson Ted M, Mao Xiaobo
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.
Mol Neurodegener. 2025 Jan 23;20(1):10. doi: 10.1186/s13024-025-00797-1.
α-Synucleinopathies constitute a spectrum of neurodegenerative disorders, including Parkinson's disease (PD), Lewy body dementia (LBD), Multiple System Atrophy (MSA), and Alzheimer's disease concurrent with LBD (AD-LBD). These disorders are unified by a pathological hallmark: aberrant misfolding and accumulation of α-synuclein (α-syn). This review delves into the pivotal role of α-syn, the key agent in α-synucleinopathy pathophysiology, and provides a survey of potential therapeutics that target cell-to-cell spread of pathologic α-syn. Recognizing the intricate complexity and multifactorial etiology of α-synucleinopathy, the review illuminates the potential of various membrane receptors, proteins, intercellular spreading pathways, and pathological agents for therapeutic interventions. While significant progress has been made in understanding α-synucleinopathy, the pursuit of efficacious treatments remains challenging. Several strategies involving decreasing α-syn production and aggregation, increasing α-syn degradation, lowering extracellular α-syn, and inhibiting cellular uptake of α-syn are presented. The paper underscores the necessity of meticulous and comprehensive investigations to advance our knowledge of α-synucleinopathy pathology and ultimately develop innovative therapeutic strategies for α-synucleinopathies.
α-突触核蛋白病是一系列神经退行性疾病,包括帕金森病(PD)、路易体痴呆(LBD)、多系统萎缩(MSA)以及合并LBD的阿尔茨海默病(AD-LBD)。这些疾病由一个病理特征统一起来:α-突触核蛋白(α-syn)的异常错误折叠和聚集。本综述深入探讨了α-syn在α-突触核蛋白病病理生理学中的关键作用,α-syn是该病病理生理过程中的关键因素,并概述了针对病理性α-syn细胞间传播的潜在治疗方法。认识到α-突触核蛋白病的复杂程度和多因素病因,本综述阐明了各种膜受体、蛋白质、细胞间传播途径和病理因素在治疗干预方面的潜力。虽然在理解α-突触核蛋白病方面已经取得了重大进展,但寻求有效的治疗方法仍然具有挑战性。本文介绍了几种策略,包括减少α-syn的产生和聚集、增加α-syn的降解、降低细胞外α-syn水平以及抑制细胞对α-syn的摄取。本文强调了进行细致全面研究的必要性,以增进我们对α-突触核蛋白病病理学的了解,并最终开发出针对α-突触核蛋白病的创新治疗策略。