Department of Chemistry, University of Pennsylvania, 231S. 34th St, Philadelphia, PA, 19104, USA.
Department of Molecular Biophysics and Biochemistry, Department of Chemical and Environmental Engineering, Yale University, 260 Whitney Avenue, New Haven, CT, 06520-8114, USA.
Nat Commun. 2018 Apr 3;9(1):1312. doi: 10.1038/s41467-018-03651-9.
Peptide mediated gain-of-toxic function is central to pathology in Alzheimer's, Parkinson's and diabetes. In each system, self-assembly into oligomers is observed and can also result in poration of artificial membranes. Structural requirements for poration and the relationship of structure to cytotoxicity is unaddressed. Here we focus on islet amyloid polypeptide (IAPP) mediated loss-of-insulin secreting cells in patients with diabetes. Newly developed methods enable structure-function enquiry to focus on intracellular oligomers composed of hundreds of IAPP. The key insights are that porating oligomers are internally dynamic, grow in discrete steps and are not canonical amyloid. Moreover, two classes of poration occur; an IAPP-specific ligand establishes that only one is cytotoxic. Toxic rescue occurs by stabilising non-toxic poration without displacing IAPP from mitochondria. These insights illuminate cytotoxic mechanism in diabetes and also provide a generalisable approach for enquiry applicable to other partially ordered protein assemblies.
肽介导的毒性功能获得是阿尔茨海默病、帕金森病和糖尿病病理的核心。在每个系统中,都观察到寡聚体的自组装,这也可能导致人工膜穿孔。穿孔的结构要求以及结构与细胞毒性的关系尚未得到解决。在这里,我们专注于糖尿病患者胰岛淀粉样多肽(IAPP)介导的胰岛素分泌细胞丧失。新开发的方法使结构-功能研究能够集中在由数百个 IAPP 组成的细胞内寡聚体上。关键的见解是,穿孔寡聚体是内部动态的,以离散的步骤生长,并且不是典型的淀粉样蛋白。此外,发生了两类穿孔;一种 IAPP 特异性配体表明只有一种是细胞毒性的。通过稳定非毒性穿孔而不将 IAPP 从线粒体中置换出来来实现毒性挽救。这些见解阐明了糖尿病中的细胞毒性机制,也为适用于其他部分有序蛋白质组装的可推广的研究方法提供了依据。