Department of Chemistry and ‡Neurosciences Institute, University of Texas at San Antonio, One UTSA Circle, TX 78249, USA.
ACS Chem Neurosci. 2013 Jul 17;4(7):1101-13. doi: 10.1021/cn400041j. Epub 2013 May 17.
The A53T genetic missense mutation of the wild-type α-synuclein (αS) protein was initially identified in Greek and Italian families with familial Parkinson's disease. Detailed understanding of the structures and the changes induced in the wild-type αS structure by the A53T mutation, as well as establishing the direct relationships between the rapid conformational changes and free energy landscapes of these intrinsically disordered fibrillogenic proteins, helps to enhance our fundamental knowledge and to gain insights into the pathogenic mechanism of Parkinson's disease. We employed extensive parallel tempering molecular dynamics simulations along with thermodynamic calculations to determine the secondary and tertiary structural properties as well as the conformational free energy surfaces of the wild-type and A53T mutant-type αS proteins in an aqueous solution medium using both implicit and explicit water models. The confined aqueous volume effect in the simulations of disordered proteins using an explicit model for water is addressed for a model disordered protein. We also assessed the stabilities of the residual secondary structure component interconversions in αS based on free energy calculations at the atomic level with dynamics using our recently developed theoretical strategy. To the best of our knowledge, this study presents the first detailed comparison of the structural properties linked directly to the conformational free energy landscapes of the monomeric wild-type and A53T mutant-type α-synuclein proteins in an aqueous solution environment. Results demonstrate that the β-sheet structure is significantly more altered than the helical structure upon A53T mutation of the monomeric wild-type αS protein in aqueous solution. The β-sheet content close to the mutation site in the N-terminal region is more abundant while the non-amyloid-β component (NAC) and C-terminal regions show a decrease in β-sheet abundance upon A53T mutation. Obtained results utilizing our new theoretical strategy show that the residual secondary structure conversion stabilities resulting in α-helix formation are not significantly affected by the mutation. Interestingly, the residual secondary structure conversion stabilities show that secondary structure conversions resulting in β-sheet formation are influenced by the A53T mutation and the most stable residual transition yielding β-sheet occurs directly from the coil structure. Long-range interactions detected between the NAC region and the N- or C-terminal regions of the wild-type αS disappear upon A53T mutation. The A53T mutant-type αS structures are thermodynamically more stable than those of the wild-type αS protein structures in aqueous solution. Overall, the higher propensity of the A53T mutant-type αS protein to aggregate in comparison to the wild-type αS protein is related to the increased β-sheet formation and lack of strong intramolecular long-range interactions in the N-terminal region in comparison to its wild-type form. The specific residual secondary structure component stabilities reported herein provide information helpful for designing and synthesizing small organic molecules that can block the β-sheet forming residues, which are reactive toward aggregation.
野生型α-突触核蛋白(αS)蛋白的 A53T 遗传错义突变最初在希腊和意大利的家族性帕金森病患者中被发现。详细了解 A53T 突变引起的野生型αS 结构的变化以及建立这些无规卷曲纤维形成蛋白的快速构象变化与自由能景观之间的直接关系,有助于增强我们的基础知识并深入了解帕金森病的发病机制。我们采用广泛的并行温度分子动力学模拟以及热力学计算,使用隐式和显式水模型,在水溶液介质中确定野生型和 A53T 突变型αS 蛋白的二级和三级结构特性以及构象自由能表面。我们针对使用显式水模型模拟无序蛋白的受限水体积效应,使用我们最近开发的理论策略,对模型无序蛋白进行了评估。我们还基于原子水平的自由能计算,使用动力学方法评估了αS 中剩余二级结构成分相互转换的稳定性,该计算方法是基于我们最近的理论策略。据我们所知,这项研究首次对单体野生型和 A53T 突变型α-突触核蛋白蛋白在水溶液环境中的构象自由能景观直接相关的结构特性进行了详细比较。结果表明,单体野生型αS 蛋白中 A53T 突变后,β-折叠结构的变化明显大于螺旋结构。N 端区域靠近突变位点的β-折叠含量更为丰富,而非淀粉样β成分(NAC)和 C 端区域的β-折叠含量则减少。利用我们的新理论策略得到的结果表明,导致α-螺旋形成的剩余二级结构转换稳定性不会受到突变的显著影响。有趣的是,剩余二级结构转换稳定性表明,导致β-折叠形成的二级结构转换受 A53T 突变影响,最稳定的剩余转换直接从卷曲结构产生β-折叠。野生型αS 中 NAC 区域与 N 或 C 端区域之间的长程相互作用在 A53T 突变后消失。A53T 突变型αS 结构在水溶液中比野生型αS 蛋白结构热力学更稳定。总体而言,与野生型αS 蛋白相比,A53T 突变型αS 蛋白在水溶液中更容易聚集,这与 N 端区域β-折叠形成增加和缺乏强的分子内长程相互作用有关。本文报道的特定剩余二级结构成分稳定性提供了有关设计和合成可以阻止形成β-折叠的反应性残基的小分子的信息,这些小分子可以阻止其聚集。