人类基因组内的 G 四链体 (G4) 已经经历了广泛的分子研究，重点关注端粒、基因启动子和重复调控序列。 G4 在调节重要的生物过程中发挥着核心作用，包括端粒维持、复制、转录和翻译。用 G4 结合配体靶向这些分子过程在抗癌治疗中具有巨大的治疗潜力，并且在治疗神经、骨骼和肌肉疾病方面也显示出前景。细菌和病毒基因组中 G4 的存在也表明 G4 结合配体可能是对抗感染的关键工具。本综述概述了 G4 结合配体的进展和应用、其拟议的作用机制、面临的挑战以及其在抗癌治疗、神经系统疾病和抗病毒活性中的应用前景。

G-quadruplexes (G4s) within the human genome have undergone extensive molecular investigation, with a strong focus on telomeres, gene promoters and repetitive regulatory sequences. G4s play central roles in regulating essential biological processes, including telomere maintenance, replication, transcription and translation. Targeting these molecular processes with G4-binding ligands holds substantial therapeutic potential in anticancer treatments and has also shown promise in treating neurological, skeletal and muscular disorders. The presence of G4s in bacterial and viral genomes also suggests that G4-binding ligands could be a critical tool in fighting infections. This review provides an overview of the progress and applications of G4-binding ligands, their proposed mechanisms of action, challenges faced and prospects for their utilization in anticancer treatments, neurological disorders and antiviral activities.