目前恶性血液病治疗以化疗为主,化疗药物的耐药导致疾病复发仍为治疗中的最大难题,并且化疗对骨髓及其他脏器具有严重的毒副作用,限制了临床治疗效果及影响患者的生活质量。DNA高甲基化在恶性血液病中广泛存在,且具有潜在的可逆性,人们以此作为治疗的靶点,研究去甲基化药物治疗恶性血液病,并有望成为一种新的治疗途径。一、DNA甲基化概念表观遗传学主要研究在DNA序列不变的情况下,转录前基因在染色质水平的结构修饰对基因功能产生影响,这种修饰可通过细胞分裂和增殖周期进行传递。DNA甲基化异常是人类肿瘤中最常见的表观遗传学改变,在基因失活、基因印记、X染色体失活、基因组内寄生序列的转录抑制等多个生物学过程中发挥重要功能[1]。所谓基因的高甲基化,是指在DNA甲基转移 At present, the treatment of hematologic malignancies is based on chemotherapy, and the drug resistance of chemotherapy drugs leads to the recurrence of the disease is still the biggest problem in the treatment. Chemotherapy has serious side effects on the bone marrow and other organs, which limits the effect of clinical treatment and affects the life of patients quality. DNA hypermethylation is widespread in hematologic malignancies and has potential reversibility. As a target of therapy, demethylation drugs can be used to treat hematologic malignancies and is expected to become a new therapeutic approach. First, the concept of DNA methylation Epigenetics Main research In the case of DNA sequences unchanged, pre-transcriptional gene chromatin level of the structural modification of gene function, this modification can be passed through the cell division and proliferation cycle . DNA methylation abnormalities are the most common epigenetic changes in human tumors and play an important role in many biological processes such as gene inactivation, gene imprinting, inactivation of the X chromosome, transcriptional repression of intragenic parasitic sequences [1 ]. The so-called gene hypermethylation, refers to the DNA methyl transfer