Kaur Upneet, Lee Jennifer C
Laboratory of Protein Conformation and Dynamics, Biochemistry and Biophysics Center, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland 20892, United States.
Acc Chem Res. 2021 Jan 19;54(2):302-310. doi: 10.1021/acs.accounts.0c00453. Epub 2021 Jan 8.
α-Synuclein (α-syn) is a key protein in the etiology of Parkinson's disease. In a disease state, α-syn accumulates as insoluble amyloid fibrils enriched in β-sheet structure. However, in its functional state, α-syn adopts an amphipathic helix upon membrane association and plays a role in synaptic vesicle docking, fusion, and clustering. In this Account, we describe our contributions made in the past decade toward developing a molecular understanding of α-syn membrane interactions, which are crucial for function and have pathological implications. Three topics are covered: α-syn membrane binding probed by neutron reflectometry (NR), the effects of membrane on α-syn amyloid formation, and interactions of α-syn with cellular membranes.NR offers a unique perspective by providing direct measurements of protein penetration depth. By the use of segmentally deuterated α-syn generated through native chemical ligation, the spatial resolution of specific membrane-bound polypeptide regions was determined by NR. Additionally, we used NR to characterize the membrane-bound complex of α-syn and glucocerebrosidase, a lysosomal hydrolase whose mutations are a common genetic risk factor for Parkinson's disease. Although phosphatidylcholine (PC) is the most abundant lipid species in mammalian cells, interactions of PC with α-syn have been largely ignored because they are substantially weaker compared with the electrostatically driven binding of negatively charged lipids. We discovered that α-syn tubulates zwitterionic PC membranes, which is likely related to its involvement in synaptic vesicle fusion by stabilization of membrane curvature. Interestingly, PC lipid tubules inhibit amyloid formation, in contrast to anionic phosphatidylglycerol lipid tubules, which stimulate protein aggregation. We also found that membrane fluidity influences the propensity of α-synuclein amyloid formation. Most recently, we obtained direct evidence of binding of α-syn to exocytic sites on intact cellular membranes using a method called cellular unroofing. This method provides direct access to the cytosolic plasma membrane. Importantly, measurements of fluorescence lifetime distributions revealed that α-syn is more conformationally dynamic at the membrane interface than previously appreciated. This exquisite responsiveness to specific lipid composition and membrane topology is important for both its physiological and pathological functions. Collectively, our work has provided insights into the effects of the chemical nature of phospholipid headgroups on the interplay among membrane remodeling, protein structure, and α-syn amyloid formation.
α-突触核蛋白(α-syn)是帕金森病病因中的关键蛋白。在疾病状态下,α-syn会积聚成富含β-折叠结构的不溶性淀粉样纤维。然而,在其功能状态下,α-syn在与膜结合时会形成两亲性螺旋,并在突触小泡对接、融合和聚集过程中发挥作用。在本综述中,我们描述了过去十年中我们在深入了解α-syn膜相互作用方面所做的贡献,这种相互作用对其功能至关重要且具有病理意义。涵盖了三个主题:通过中子反射率(NR)探测α-syn膜结合、膜对α-syn淀粉样蛋白形成的影响以及α-syn与细胞膜的相互作用。NR通过直接测量蛋白质渗透深度提供了独特的视角。通过使用通过天然化学连接产生的分段氘代α-syn,特定膜结合多肽区域的空间分辨率由NR确定。此外,我们使用NR来表征α-syn与葡糖脑苷脂酶的膜结合复合物,葡糖脑苷脂酶是一种溶酶体水解酶,其突变是帕金森病常见的遗传风险因素。尽管磷脂酰胆碱(PC)是哺乳动物细胞中含量最丰富的脂质种类,但PC与α-syn的相互作用在很大程度上被忽视了,因为与带负电荷脂质的静电驱动结合相比,它们的相互作用要弱得多。我们发现α-syn能使两性离子PC膜形成微管,这可能与其通过稳定膜曲率参与突触小泡融合有关。有趣的是,PC脂质微管抑制淀粉样蛋白形成,而阴离子磷脂酰甘油脂质微管则刺激蛋白质聚集。我们还发现膜流动性会影响α-突触核蛋白淀粉样蛋白形成的倾向。最近,我们使用一种称为细胞去顶的方法获得了α-syn与完整细胞膜上胞吐位点结合的直接证据。这种方法可直接接触胞质质膜。重要的是,荧光寿命分布测量表明,α-syn在膜界面处的构象比以前认为的更具动态性。这种对特定脂质组成和膜拓扑结构的精确响应性对其生理和病理功能都很重要。总的来说,我们的工作为磷脂头部基团的化学性质对膜重塑、蛋白质结构和α-syn淀粉样蛋白形成之间相互作用的影响提供了见解。