Gower Carrie M, Thomas Jason R, Harrington Edmund, Murphy Jason, Chang Matthew E K, Cornella-Taracido Ivan, Jain Rishi K, Schirle Markus, Maly Dustin J
Novartis Institutes for Biomedical Research , Cambridge, Massachusetts 02139, United States.
ACS Chem Biol. 2016 Jan 15;11(1):121-31. doi: 10.1021/acschembio.5b00847. Epub 2015 Nov 6.
Loss-of-function studies are valuable for elucidating kinase function and the validation of new drug targets. While genetic techniques, such as RNAi and genetic knockouts, are highly specific and easy to implement, in many cases post-translational perturbation of kinase activity, specifically pharmacological inhibition, is preferable. However, due to the high degree of structural similarity between kinase active sites and the large size of the kinome, identification of pharmacological agents that are sufficiently selective to probe the function of a specific kinase of interest is challenging, and there is currently no systematic method for accomplishing this goal. Here, we present a modular chemical genetic strategy that uses antibody mimetics as highly selective targeting components of bivalent kinase inhibitors. We demonstrate that it is possible to confer high kinase selectivity to a promiscuous ATP-competitive inhibitor by tethering it to an antibody mimetic fused to the self-labeling protein SNAPtag. With this approach, a potent bivalent inhibitor of the tyrosine kinase Abl was generated. Profiling in complex cell lysates, with competition-based quantitative chemical proteomics, revealed that this bivalent inhibitor possesses greatly enhanced selectivity for its target, BCR-Abl, in K562 cells. Importantly, we show that both components of the bivalent inhibitor can be assembled in K562 cells to block the ability of BCR-Abl to phosphorylate a direct cellular substrate. Finally, we demonstrate the generality of using antibody mimetics as components of bivalent inhibitors by generating a reagent that is selective for the activated state of the serine/threonine kinase ERK2.
功能丧失研究对于阐明激酶功能和验证新的药物靶点具有重要价值。虽然RNA干扰和基因敲除等基因技术具有高度特异性且易于实施,但在许多情况下,激酶活性的翻译后扰动,特别是药理学抑制,更为可取。然而,由于激酶活性位点之间的高度结构相似性以及激酶组的庞大规模,鉴定出足够选择性以探究特定感兴趣激酶功能的药理学试剂具有挑战性,目前尚无实现这一目标的系统方法。在此,我们提出一种模块化化学遗传策略,该策略使用抗体模拟物作为二价激酶抑制剂的高度选择性靶向成分。我们证明,通过将一种混杂的ATP竞争性抑制剂与融合到自标记蛋白SNAPtag的抗体模拟物相连,可以赋予其对激酶的高选择性。采用这种方法,生成了一种有效的酪氨酸激酶Abl二价抑制剂。基于竞争的定量化学蛋白质组学在复杂细胞裂解物中的分析表明,这种二价抑制剂在K562细胞中对其靶点BCR-Abl具有大大增强的选择性。重要的是,我们表明二价抑制剂的两个成分都可以在K562细胞中组装,以阻断BCR-Abl磷酸化直接细胞底物的能力。最后,我们通过生成一种对丝氨酸/苏氨酸激酶ERK2的活化状态具有选择性的试剂,证明了使用抗体模拟物作为二价抑制剂成分的通用性。