Univ. de Bordeaux, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France.
CNRS, Institut des Maladies Neurodégénératives, UMR 5293, F-33000 Bordeaux, France.
Biomolecules. 2020 Mar 3;10(3):391. doi: 10.3390/biom10030391.
Parkinson's Disease (PD) is characterized both by the loss of dopaminergic neurons in the substantia nigra and the presence of cytoplasmic inclusions called Lewy Bodies. These Lewy Bodies contain the aggregated α-synuclein (α-syn) protein, which has been shown to be able to propagate from cell to cell and throughout different regions in the brain. Due to its central role in the pathology and the lack of a curative treatment for PD, an increasing number of studies have aimed at targeting this protein for therapeutics. Here, we reviewed and discussed the many different approaches that have been studied to inhibit α-syn accumulation via direct and indirect targeting. These analyses have led to the generation of multiple clinical trials that are either completed or currently active. These clinical trials and the current preclinical studies must still face obstacles ahead, but give hope of finding a therapy for PD with time.
帕金森病（PD）的特征是黑质中多巴胺能神经元的丧失和称为路易体的细胞质包含物的存在。这些路易体包含聚集的α-突触核蛋白（α-syn），已经表明它能够在细胞间和大脑的不同区域传播。由于其在病理学中的核心作用以及缺乏针对 PD 的治愈性治疗方法，越来越多的研究旨在针对这种蛋白质进行治疗。在这里，我们回顾和讨论了许多不同的方法，这些方法旨在通过直接和间接靶向来抑制α-syn 的积累。这些分析导致了多个临床试验的完成或正在进行中。这些临床试验和当前的临床前研究仍然必须面对未来的障碍，但随着时间的推移，为 PD 找到一种治疗方法带来了希望。
