Howard Hughes Medical Institute, Stanford University School of Medicine, Stanford, California 94305
Department of Molecular and Cellular Physiology, Stanford University School of Medicine, Stanford, California 94305.
J Biol Chem. 2019 Sep 20;294(38):13876-13886. doi: 10.1074/jbc.RA119.009213. Epub 2019 Aug 6.
Ligand-induced dimerization is the predominant mechanism through which secreted proteins activate cell surface receptors to transmit essential biological signals. Cytokines are a large class of soluble proteins that dimerize transmembrane receptors into precise signaling topologies, but there is a need for alternative, engineerable ligand scaffolds that specifically recognize and stabilize these protein interactions. Recombinant antibodies can potentially serve as robust and versatile platforms for cytokine complex stabilization, and their specificity allows for tunable modulation of dimerization equilibrium. Here, we devised an evolutionary strategy to isolate monovalent antibody fragments that bridge together two different receptor subunits in a cytokine-receptor complex, precisely as the receptors are disposed in their natural signaling orientations. To do this, we screened a naive antibody library against a stabilized ligand-receptor ternary complex that acted as a "molecular cast" of the natural receptor dimer conformation. Our selections elicited "stapler" single-chain variable fragments (scFvs) of antibodies that specifically engage the interleukin-4 receptor heterodimer. The 3.1 Å resolution crystal structure of one such stapler revealed that, as intended, this scFv recognizes a composite epitope between the two receptors as they are positioned in the complex. Extending our approach, we evolved a stapler scFv that specifically binds to and stabilizes the interface between the interleukin-2 cytokine and one of its receptor subunits, leading to a 15-fold enhancement in interaction affinity. This demonstration that scFvs can be selected to recognize epitopes that span protein interfaces presents new opportunities to engineer structurally defined antibodies for a broad range of research and therapeutic applications.
配体诱导的二聚化是分泌蛋白激活细胞表面受体以传递重要生物信号的主要机制。细胞因子是一大类可溶性蛋白,它们将跨膜受体二聚化成精确的信号拓扑结构,但需要替代的、可工程设计的配体支架,这些支架能够特异性识别和稳定这些蛋白质相互作用。重组抗体可以作为细胞因子复合物稳定的强大而多功能的平台,并且它们的特异性允许对二聚化平衡进行可调谐的调节。在这里,我们设计了一种进化策略来分离单价抗体片段,这些片段在细胞因子受体复合物中将两个不同的受体亚基桥接在一起,就像受体在其自然信号取向中那样。为此,我们针对稳定的配体-受体三元复合物筛选了一个原始抗体文库,该复合物充当了自然受体二聚体构象的“分子铸模”。我们的选择引出了“订书钉”单链可变片段(scFv)的抗体,这些抗体特异性地与白细胞介素-4 受体异二聚体结合。这种订书钉 scFv 的分辨率为 3.1 Å 的晶体结构表明,正如预期的那样,该 scFv 识别两个受体在复合物中定位时的复合表位。通过扩展我们的方法,我们进化出一种订书钉 scFv,该 scFv 特异性地结合并稳定白细胞介素-2 细胞因子与其受体亚基之一之间的界面,从而使相互作用亲和力提高了 15 倍。该证明 scFv 可以被选择来识别跨越蛋白质界面的表位,为广泛的研究和治疗应用提供了设计结构定义抗体的新机会。