虽然端粒酶在正常分化的人体组织中保持沉默，但在大多数人类癌症中会重新激活。因此，端粒酶几乎是普遍的肿瘤学靶标。本更新描述了使用多种靶向端粒酶方法的临床前和临床进展。这些包括直接端粒酶抑制剂、G-四链体 DNA 相互作用配体、基于端粒酶的疫苗平台、端粒酶启动子驱动的减毒病毒以及端粒酶介导的端粒靶向方法。虽然 imetelstat 最近已获得美国食品和药物管理局 (FDA) 的批准，但其他几种方法正处于后期临床开发阶段。我们将回顾主要方法的优缺点。版权所有 © 2024 Cold Spring Harbor Laboratory Press；版权所有。

While silent in normal differentiated human tissues, telomerase is reactivated in most human cancers. Thus, telomerase is an almost universal oncology target. This update describes preclinical and clinical advancements using a variety of approaches to target telomerase. These include direct telomerase inhibitors, G-quadruplex DNA-interacting ligands, telomerase-based vaccine platforms, telomerase promoter-driven attenuated viruses, and telomerase-mediated telomere targeting approaches. While imetelstat has been recently approved by the Food and Drug Administration (FDA), several other approaches are in late-stage clinical development. The pros and cons of the major approaches will be reviewed.Copyright © 2024 Cold Spring Harbor Laboratory Press; all rights reserved.