The Skaggs Institute for Chemical Biology, The Scripps Research Institute, 10550 North Torrey Pines Road, La Jolla, CA 92037, USA.
Proc Natl Acad Sci U S A. 2010 Dec 7;107(49):20941-6. doi: 10.1073/pnas.1011663107. Epub 2010 Nov 17.
Serine hydrolases (SHs) are one of the largest and most diverse enzyme classes in mammals. They play fundamental roles in virtually all physiological processes and are targeted by drugs to treat diseases such as diabetes, obesity, and neurodegenerative disorders. Despite this, we lack biological understanding for most of the 110+ predicted mammalian metabolic SHs, in large part because of a dearth of assays to assess their biochemical activities and a lack of selective inhibitors to probe their function in living systems. We show here that the vast majority (> 80%) of mammalian metabolic SHs can be labeled in proteomes by a single, active site-directed fluorophosphonate probe. We exploit this universal activity-based assay in a library-versus-library format to screen 70+ SHs against 140+ structurally diverse carbamates. Lead inhibitors were discovered for ∼40% of the screened enzymes, including many poorly characterized SHs. Global profiles identified carbamate inhibitors that discriminate among highly sequence-related SHs and, conversely, enzymes that share inhibitor sensitivity profiles despite lacking sequence homology. These findings indicate that sequence relatedness is not a strong predictor of shared pharmacology within the SH superfamily. Finally, we show that lead carbamate inhibitors can be optimized into pharmacological probes that inactivate individual SHs with high specificity in vivo.
丝氨酸水解酶(SHs)是哺乳动物中最大和最多样化的酶类之一。它们在几乎所有生理过程中都发挥着基本作用,并且是用于治疗糖尿病、肥胖症和神经退行性疾病等疾病的药物的靶标。尽管如此,我们对 110 多种预测的哺乳动物代谢 SH 中的大多数仍缺乏生物学理解,这在很大程度上是因为缺乏评估其生化活性的测定方法和用于在活系统中探测其功能的选择性抑制剂。我们在这里表明,绝大多数 (>80%)哺乳动物代谢 SH 可以通过单个活性位点定向氟膦酸盐探针在蛋白质组中标记。我们以文库对文库的格式利用这种通用的基于活性的测定方法,用 140 多种结构多样的氨基甲酸酯对 70 多种 SH 进行筛选。针对约 40%筛选出的酶发现了先导抑制剂,其中包括许多未充分表征的 SH。确定的氨基甲酸酯抑制剂的全局图谱可区分高度序列相关的 SH,反之,尽管缺乏序列同源性,但具有抑制剂敏感性图谱的酶也是如此。这些发现表明,序列相似性并不是 SH 超家族中共享药理学的有力预测因素。最后,我们表明,先导氨基甲酸酯抑制剂可以优化为药理学探针,这些探针可以在体内高度特异性地使单个 SH 失活。