Department of Pharmacology, Vanderbilt University, Nashville, TN, United States.
Department of Pharmacology, Vanderbilt University, Nashville, TN, United States.
Prog Mol Biol Transl Sci. 2019;161:29-45. doi: 10.1016/bs.pmbts.2018.09.004. Epub 2018 Oct 24.
Arrestins play a key role in homologous desensitization of G protein-coupled receptors (GPCRs) and regulate several other vital signaling pathways in cells. Considering the critical roles of these proteins in cellular signaling, surprisingly few disease-causing mutations in human arrestins were described. Most of these are loss-of-function mutations of visual arrestin-1 that cause excessive rhodopsin signaling and hence night blindness. Only one dominant arrestin-1 mutation was discovered so far. It reduces the thermal stability of the protein, which likely results in photoreceptor death via unfolded protein response. In case of the two nonvisual arrestins, only polymorphisms were described, some of which appear to be associated with neurological disorders and altered response to certain treatments. Structure-function studies revealed several ways of enhancing arrestins' ability to quench GPCR signaling. These enhanced arrestins have potential as tools for gene therapy of disorders associated with excessive signaling of mutant GPCRs.
抑制蛋白在 G 蛋白偶联受体 (GPCR) 的同源脱敏中起着关键作用,并调节细胞中的几个其他重要信号通路。考虑到这些蛋白质在细胞信号转导中的关键作用,出人意料的是,在人类抑制蛋白中发现的致病突变很少。大多数这些是视觉抑制蛋白-1 的功能丧失突变,导致过度的视蛋白信号,因此导致夜盲症。迄今为止,只发现了一个显性抑制蛋白-1 突变。它降低了蛋白质的热稳定性,这可能通过未折叠蛋白反应导致光感受器死亡。在两种非视觉抑制蛋白的情况下,只描述了多态性,其中一些似乎与神经紊乱和对某些治疗的反应改变有关。结构功能研究揭示了几种增强抑制蛋白抑制 GPCR 信号能力的方法。这些增强的抑制蛋白有可能作为治疗与突变 GPCR 过度信号相关的疾病的基因治疗工具。