DNA甲基化是调节基因表达而不改变DNA碱基序列的表观遗传修饰，通过沉默肿瘤抑制基因在癌症发展中发挥关键作用。DNA去甲基化药物在临床上已经显示出疗效，然而，高效性和特异性的DNA去甲基化药物尚未出现。目前，在市场上已有2种药物阿扎胞苷和地西他滨用于治疗骨髓增生异常综合征。寻找直接结合靶点新的抑制剂是未来的方向。从抗肿瘤活性和临床研究方面介绍了DNA去甲基化药物的研究进展。

DNA methylation is an epigenetic modification that modulates gene expression without altering the DNA base sequence, which plays a crucial role in cancer by silencing tumor suppressor genes. DNA demethylating agents have shown efficacy in clinics. However, highly efficient and specific DNA demethylating agents have not yet been found. At present, two drugs, azacitidine and decitabine are on the market for the treatment of myelodysplastic syndrome. Improving methods will certainly lead to the prediction of novel directly binding inhibitors in the future. This article reviewed the progress on antitumor activities and clinical studies of DNA demethylating agents.

中国博士后基金资助项目（2016M591399）；福建省中青年教师教育科研项目（JAT160852）；天津市自然科学基金重点项目（16JCZDJC32600）