Lee Eunyoung, Koh Youngil, Hong Junshik, Eom Hyeon Seok, Yoon Sung Soo
Department of Internal Medicine, Center for Hematologic Malignancy, National Cancer Center, Goyang, Korea.
Department of Internal Medicine, Seoul National University Hospital, Cancer Research Institute, Seoul National University College of Medicine, Seoul, Korea.
J Korean Med Sci. 2021 Apr 5;36(13):e85. doi: 10.3346/jkms.2021.36.e85.
Acute myeloid leukemia (AML) is a complicated disease characterized by genetic heterogeneity and simultaneous alterations in multiple genes. For decades, its only curative method has been intensive induction chemotherapy with or without allogeneic hematopoietic stem cell transplantation, and this approach cannot be applied to elderly patients, who make up more than 50% of AML patients. Recent advances in genomics facilitated the elucidation of various mutations related to AML, and the most frequent mutations were discovered in epigenetic regulators. Alterations to epigenetic modifications that are essential for normal cell biology, including DNA methylation and histone acetylation, have been identified. As epigenetic dysregulation is an important carcinogenic mechanism and some epigenetic changes are reversible, these epigenetic alterations have become targets for novel drug development against AML. This review summarizes the recent advances in epigenetic therapies for AML and discusses future research directions.
急性髓系白血病（AML）是一种复杂的疾病，其特征在于基因异质性和多个基因的同时改变。几十年来，其唯一的治愈方法一直是强化诱导化疗，无论是否进行异基因造血干细胞移植，而这种方法不适用于占AML患者50%以上的老年患者。基因组学的最新进展有助于阐明与AML相关的各种突变，并且在表观遗传调节因子中发现了最常见的突变。已经确定了对正常细胞生物学至关重要的表观遗传修饰的改变，包括DNA甲基化和组蛋白乙酰化。由于表观遗传失调是一种重要的致癌机制，并且一些表观遗传变化是可逆的，这些表观遗传改变已成为针对AML的新型药物开发的靶点。本综述总结了AML表观遗传治疗的最新进展，并讨论了未来的研究方向。
