Pharmacology Graduate Group, Raymond and Ruth Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, 19104, USA.
Department of Biological Sciences, University of Alabama, Tuscaloosa, AL, 35487, USA.
Sci Rep. 2019 Feb 27;9(1):2937. doi: 10.1038/s41598-019-39480-z.
Growing evidence implicates α-synuclein aggregation as a key driver of neurodegeneration in Parkinson's disease (PD) and other neurodegenerative disorders. Herein, the molecular and structural mechanisms of inhibiting α-synuclein aggregation by novel analogs of nordihydroguaiaretic acid (NDGA), a phenolic dibenzenediol lignan, were explored using an array of biochemical and biophysical methodologies. NDGA analogs induced modest, progressive compaction of monomeric α-synuclein, preventing aggregation into amyloid-like fibrils. This conformational remodeling preserved the dynamic adoption of α-helical conformations, which are essential for physiological membrane interactions. Oxidation-dependent NDGA cyclization was required for the interaction with monomeric α-synuclein. NDGA analog-pretreated α-synuclein did not aggregate even without NDGA-analogs in the aggregation mixture. Strikingly, NDGA-pretreated α-synuclein suppressed aggregation of naïve untreated aggregation-competent monomeric α-synuclein. Further, cyclized NDGA reduced α-synuclein-driven neurodegeneration in Caenorhabditis elegans. The cyclized NDGA analogs may serve as a platform for the development of small molecules that stabilize aggregation-resistant α-synuclein monomers without interfering with functional conformations yielding potential therapies for PD and related disorders.
越来越多的证据表明,α-突触核蛋白聚集是帕金森病 (PD) 和其他神经退行性疾病神经变性的关键驱动因素。在此,通过使用一系列生化和生物物理方法,研究了新型北美圣草次酸 (NDGA) 类似物抑制 α-突触核蛋白聚集的分子和结构机制。NDGA 类似物诱导单体 α-突触核蛋白适度、渐进地凝聚,防止其聚集成类淀粉样原纤维。这种构象重塑保留了对生理膜相互作用至关重要的 α-螺旋构象的动态采用。与单体 α-突触核蛋白的相互作用需要依赖氧化的 NDGA 环化。即使在聚集混合物中没有 NDGA 类似物,经 NDGA 类似物预处理的 α-突触核蛋白也不会聚集。值得注意的是,经 NDGA 预处理的 α-突触核蛋白抑制了未处理的聚集能力强的单体 α-突触核蛋白的聚集。此外,环化的 NDGA 减少了秀丽隐杆线虫中 α-突触核蛋白驱动的神经变性。环化的 NDGA 类似物可以作为开发稳定抗聚集 α-突触核蛋白单体的小分子的平台,而不干扰产生 PD 和相关疾病潜在治疗方法的功能构象。