Saleh Ibrahim G, Shimogawa Marie, Ramirez Jennifer, Abakah Bernard, Venkatesh Yarra, James Honey Priya, Li Ming-Hao, Louie Sarah A, Lougee Marshall G, Chia Wai-Kit, Brue Christopher, Cooley Richard B, Mehl Ryan A, Baumgart Tobias, Mach Robert H, Eliezer David, Rhoades Elizabeth, Petersson E James
Department of Chemistry, School of Arts and Sciences, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Graduate Group in Biochemistry, Biophysics, and Chemical Biology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Protein Sci. 2025 Oct;34(10):e70302. doi: 10.1002/pro.70302.
Alpha-synuclein (αS) and tau play important roles in the pathology of Parkinson's disease and Alzheimer's disease, respectively, as well as numerous other neurodegenerative diseases. Both proteins are classified as intrinsically disordered proteins (IDPs), as they have no stable structure that underlies their function in healthy tissue, and both proteins are prone to aggregation in disease states. There is substantial interest in understanding the roles that post-translational modifications (PTMs) play in regulating the structural dynamics and function of αS and tau monomers, as well as their propensity to aggregate. While there have been many valuable insights into site-specific effects of PTMs garnered through chemical synthesis and semi-synthesis, these techniques are often outside of the expertise of biochemistry and biophysics laboratories wishing to study αS and tau. Therefore, we have assembled a primer on genetic code expansion and enzymatic modification approaches to installing PTMs into αS and tau site-specifically, including isotopic labeling for NMR and fluorescent labeling for biophysics and microscopy experiments. These methods should be enabling for those wishing to study authentic PTMs in αS or tau as well as the broader field of IDPs and aggregating proteins.
α-突触核蛋白(αS)和tau蛋白分别在帕金森病和阿尔茨海默病以及许多其他神经退行性疾病的病理过程中发挥重要作用。这两种蛋白质都被归类为内在无序蛋白(IDP),因为它们在健康组织中没有稳定的结构来支撑其功能,并且在疾病状态下都容易聚集。人们对理解翻译后修饰(PTM)在调节αS和tau单体的结构动力学和功能以及它们聚集倾向方面所起的作用非常感兴趣。虽然通过化学合成和半合成已经获得了许多关于PTM位点特异性效应的宝贵见解,但这些技术通常超出了希望研究αS和tau的生物化学和生物物理实验室的专业范围。因此,我们编写了一份关于遗传密码扩展和酶促修饰方法的入门指南,用于将PTM位点特异性地安装到αS和tau中,包括用于核磁共振(NMR)的同位素标记以及用于生物物理和显微镜实验的荧光标记。这些方法应该能够帮助那些希望研究αS或tau中真实PTM的人以及更广泛的内在无序蛋白和聚集蛋白领域的研究人员。