沃纳解旋酶作为错配修复缺陷型结直肠癌的治疗靶点。
Werner helicase as a therapeutic target in mismatch repair deficient colorectal cancer.
作者信息
Hao Suisui, Liu Zhaojin, Lenz Heinz-Josef, Yu Jian, Zhang Lin
机构信息
Department of Medicine, Keck School of Medicine of University of Southern California (USC), Los Angeles, CA 90033, USA; Norris Comprehensive Cancer Center, Keck School of Medicine of USC, Los Angeles, CA 90033, USA.
Department of Medicine, Keck School of Medicine of University of Southern California (USC), Los Angeles, CA 90033, USA; Norris Comprehensive Cancer Center, Keck School of Medicine of USC, Los Angeles, CA 90033, USA.
出版信息
DNA Repair (Amst). 2025 May;149:103831. doi: 10.1016/j.dnarep.2025.103831. Epub 2025 Apr 3.
Colorectal cancer (CRC) is one of the leading causes of cancer-related deaths in the United States. A key driver of CRC development is microsatellite instability (MSI), which is caused by DNA mismatch repair deficiency and characterized by hypermutability of short-tandem repeat sequences. A significant portion of MSI CRCs do not respond to checkpoint immunotherapy treatments, highlighting an unmet need for improved therapies. Recent studies have revealed that MSI cancer cells require Werner (WRN), a RecQ family DNA helicase, for survival. Inhibiting WRN has emerged as a promising approach for targeting MSI CRCs that are insensitive to standard therapies. Several highly potent small-molecule WRN inhibitors have been developed and exhibited striking in vitro and in vivo activities against MSI cancers. Two of these WRN inhibitors, HRO761 and VVD-133214, have recently entered clinical trials. In this review, we summarize recent studies on WRN as a synthetic lethal target in MSI CRC and the development of WRN inhibitors as a new class of anticancer agents.
结直肠癌(CRC)是美国癌症相关死亡的主要原因之一。CRC发展的一个关键驱动因素是微卫星不稳定性(MSI),它由DNA错配修复缺陷引起,其特征是短串联重复序列的高突变性。相当一部分MSI CRC对检查点免疫疗法无反应,这突出表明对改进疗法存在未满足的需求。最近的研究表明,MSI癌细胞需要Werner(WRN),一种RecQ家族DNA解旋酶来维持生存。抑制WRN已成为靶向对标准疗法不敏感的MSI CRC的一种有前景的方法。已经开发出几种高效的小分子WRN抑制剂,并在体外和体内对MSI癌症表现出显著活性。其中两种WRN抑制剂HRO761和VVD - 133214最近已进入临床试验。在这篇综述中,我们总结了关于WRN作为MSI CRC中合成致死靶点的最新研究以及WRN抑制剂作为一类新型抗癌药物的开发情况。