Department of Pathology, Stanford University School of Medicine, Stanford, California 94305, USA.
J Am Chem Soc. 2013 Jun 19;135(24):9130-8. doi: 10.1021/ja403521u. Epub 2013 Jun 6.
Caspases are cysteine proteases that play essential roles in apoptosis and inflammation. Unfortunately, their highly conserved active sites and overlapping substrate specificities make it difficult to use inhibitors or activity-based probes to study the function, activation, localization, and regulation of individual members of this family. Here we describe a strategy to engineer a caspase to contain a latent nucleophile that can be targeted by a probe containing a suitably placed electrophile, thereby allowing specific, irreversible inhibition and labeling of only the engineered protease. To accomplish this, we have identified a non-conserved residue on the small subunit of all caspases that is near the substrate-binding pocket and that can be mutated to a non-catalytic cysteine residue. We demonstrate that an active-site probe containing an irreversible binding acrylamide electrophile can specifically target this cysteine residue. Here we validate the approach using the apoptotic mediator, caspase-8, and the inflammasome effector, caspase-1. We show that the engineered enzymes are functionally identical to the wild-type enzymes and that the approach allows specific inhibition and direct imaging of the engineered targets in cells. Therefore, this method can be used to image localization and activation as well as the functional contributions of individual caspase proteases to the process of cell death or inflammation.
半胱天冬氨酸蛋白酶(caspases)是一类在细胞凋亡和炎症中发挥重要作用的半胱氨酸蛋白酶。不幸的是,由于它们的高度保守的活性位点和重叠的底物特异性,使用抑制剂或基于活性的探针来研究该家族中各个成员的功能、激活、定位和调控变得非常困难。在这里,我们描述了一种工程化 caspase 的策略,该策略使 caspase 包含一个潜伏的亲核基团,该亲核基团可以被含有适当放置的亲电基团的探针靶向,从而可以特异性、不可逆地抑制和标记仅工程化的蛋白酶。为了实现这一点,我们已经在所有 caspases 的小亚基上鉴定出一个非保守残基,该残基位于底物结合口袋附近,并且可以突变为非催化半胱氨酸残基。我们证明了含有不可逆结合丙烯酰胺亲电基团的活性位点探针可以特异性地靶向该半胱氨酸残基。在这里,我们使用凋亡介质 caspase-8 和炎症体效应物 caspase-1 验证了该方法。我们表明,工程化的酶在功能上与野生型酶相同,并且该方法允许在细胞中特异性抑制和直接成像工程化的靶标。因此,该方法可用于成像定位和激活以及单个半胱天冬氨酸蛋白酶对细胞死亡或炎症过程的功能贡献。