以生物靶分子为基础进行抗肿瘤药物先导化合物的筛选是抗肿瘤药物研究的热点之一.DNAG-四链体结构的发现和现代分子生物学技术对其与癌症关系的揭示,为目前抗肿瘤药物研发提供了一个新的契机.能够诱导DNA形成G-四链体结构或者与G-四链体特异性结合并使之稳定的化合物有望抑制肿瘤细胞的生长,从而达到抗癌的作用.以G-四链体为抗癌药物作用靶点对化合物进行筛选和结构设计是目前化学家和生物学家的关注点.本文旨在针对靶向G-四链体的抗肿瘤分子筛选、结构设计以及抗肿瘤药物开发三个方面的最新研究进展进行综述.首先,介绍两种基于G-四链体对其配体结构特异性识别而对化合物进行结构筛选的研究方法:基于核磁共振进行抗肿瘤化合物筛选方法以及计算机虚拟筛选.其次,从化合物与G-四链体之间静电相互作用方面来进行G-四链体配体的结构设计,主要包括以下4种类型:(1)原位胺的质子化;(2)通过杂环芳香族化合物上的N-甲基化;(3)中心金属离子的存在;(4)不带电荷的化合物.最后,对目前基于G-四链体为抗肿瘤作用靶点、已经走向临床实验的CX-3543,AS1411两个抗肿瘤药物的开发与作用机制进行介绍. The screening of anticancer drug lead compounds on the basis of biological target molecules is one of the hot spots in the field of anticancer drugs.Discovery of the structure of DNA G-quadruplex and the revelation of modern molecular biology technology to the cancer reveal that antitumor Drug discovery provides a new opportunity to be able to induce DNA formation of G-quadruplex structure or with G-quadruplex-specific and stable compound is expected to inhibit tumor cell growth, so as to achieve the role of anti-cancer. G-quadruplex is a target of anticancer drug screening and structural design of compounds is the focus of chemists and biologists.This paper aims to target G-quadruplex antitumor molecular screening, structural design As well as the development of anti-tumor drugs in recent years.Firstly, two kinds of research methods for structural screening of compounds based on G-quadruplexespecific recognition of their ligands are introduced: anti-tumor based on nuclear magnetic resonance Compound screening method and computer virtual screening.Secondly, the structural design of G-quadruplex ligand was carried out in terms of the electrostatic interaction between the compound and G-quadruplex, Mainly include the following four types: (1) protonation of amine in situ; (2) N-methylation on heterocyclic aromatic compounds; (3) presence of central metal ion; (4) uncharged Compounds.Finally, the development and mechanism of two anti-tumor drugs, CX-3543 and AS1411, which have been going to clinical trials based on G-quadruplex as antitumor target, are introduced.