Department of Biochemistry and Molecular Biology, College of Medicine, University of Florida, 1600 SW Archer Rd., PO Box 100245, Gainesville, FL 32610, USA.
Bioorg Med Chem. 2013 Mar 15;21(6):1526-33. doi: 10.1016/j.bmc.2012.08.019. Epub 2012 Aug 28.
Carbonic anhydrases (CAs, EC 4.2.1.1) are a group of metalloenzymes that play important roles in carbon metabolism, pH regulation, CO2 fixation in plants, ion transport etc., and are found in all eukaryotic and many microbial organisms. This family of enzymes catalyzes the interconversion of CO2 and HCO3(-). There are at least 16 different CA isoforms in the alpha structural class (α-CAs) that have been isolated in higher vertebrates, with CA isoform II (CA II) being ubiquitously abundant in all human cell types. CA inhibition has been exploited clinically for decades for various classes of diuretics and anti-glaucoma treatment. The characterization of the overexpression of CA isoform IX (CA IX) in certain tumors has raised interest in CA IX as a diagnostic marker and drug target for aggressive cancers and therefore the development of CA IX specific inhibitors. An important goal in the field of CA is to identify, rationalize, and design potential compounds that will preferentially inhibit CA IX over all other isoforms of CA. The variations in the active sites between isoforms of CA are subtle and this causes non-specific CA inhibition which leads to various side effects. In the case of CA IX inhibition, CA II along with other isoforms of CA provide off-target binding sites which is undesirable for cancer treatment. The focus of this article is on CA IX inhibition and two different structural approaches to CA isoform specific drug designing: tail approach and fragment addition approach.
碳酸酐酶(CA,EC 4.2.1.1)是一组金属酶,在碳代谢、pH 调节、植物中的 CO2 固定、离子转运等方面发挥重要作用,存在于所有真核生物和许多微生物中。该酶家族催化 CO2 和 HCO3(-) 的相互转化。在高等脊椎动物中已经分离出至少 16 种不同的α结构类碳酸酐酶(α-CAs)同工型,其中同工型 II(CA II)在所有人类细胞类型中普遍丰富。几十年来,CA 抑制已被用于各种类别的利尿剂和抗青光眼治疗的临床应用。某些肿瘤中 CA 同工型 IX(CA IX)的过表达特征引起了人们对 CA IX 作为诊断标志物和侵袭性癌症药物靶点的兴趣,因此开发了 CA IX 特异性抑制剂。CA 领域的一个重要目标是识别、合理化和设计潜在化合物,这些化合物将优先抑制 CA IX 而不是 CA 的所有其他同工型。CA 同工型之间的活性位点变化很细微,这导致非特异性 CA 抑制,从而导致各种副作用。在 CA IX 抑制的情况下,CA II 与 CA 的其他同工型一起提供非靶标结合位点,这对于癌症治疗是不理想的。本文的重点是 CA IX 抑制和两种不同的 CA 同工型特异性药物设计结构方法:尾部方法和片段添加方法。