Department of Chemistry and Biochemistry, University of California, San Diego, La Jolla, California 92093-0358, United States.
ACS Chem Biol. 2020 Mar 20;15(3):669-674. doi: 10.1021/acschembio.9b00833. Epub 2020 Feb 17.
While recognized as a therapeutic target, the spliceosome may offer a robust vector to improve established therapeutics against other protein targets. Here, we describe how modulating the spliceosome using small molecule splice modulators (SPLMs) can prime a cell for sensitivity to a target-specific drug. Using the cell cycle regulators aurora kinase and polo-like kinase as models, this study demonstrates how the combination of SPLM treatment in conjunction with kinase inhibition offers synergy for antitumor activity using reduced, sublethal levels of SPLM and kinase inhibitors. This concept of splice-modulated drug attenuation suggests a possible approach to enhance therapeutic agents that have shown limited applicability due to high toxicity or low efficacy.
虽然剪接体已被确认为治疗靶点,但它也可能成为一个强大的载体,用于改进针对其他蛋白质靶点的现有治疗方法。在这里,我们描述了如何使用小分子剪接调节剂(SPLM)来调节剪接体,从而使细胞对特定药物的敏感性增加。本研究以细胞周期调节剂 Aurora 激酶和 Polo 样激酶为模型,证明了 SPLM 联合激酶抑制剂治疗如何通过降低 SPLM 和激酶抑制剂的亚致死浓度来实现抗肿瘤活性的协同作用。这种剪接调节药物衰减的概念提示了一种可能的方法,用于增强因高毒性或低疗效而应用受限的治疗药物。