Epigenetic therapy for leukemias: a comprehensive review from biological markers to practice

Leukemia comprises a group of malignant clonal hematopoietic stem cell disorders where epigenetic abnormalities significantly contribute to its pathogenesis and progression. Epigenetic alterations, such as DNA methylation, histone modifications, and non-coding RNA dysregulation, affect gene expression and cellular function, driving leukemia development. In recent years, therapeutic strategies targeting these epigenetic abnormalities have emerged as a vibrant area of research. DNA methylation is one of the common epigenetic modifications in leukemia. DNA methyltransferase inhibitors (e.g., azidothymidine) are able to reverse aberrant DNA methylation patterns and reactivate silenced oncogenes, thereby inhibiting the proliferation of leukemia cells and inducing apoptosis. Histone acetylase inhibitors and histone methylase inhibitors regulate the acetylation and methylation status of histones, affecting gene expression and cell cycle progression. These drugs inhibit the malignant proliferation and induce differentiation or apoptosis of leukemia cells by altering their epigenetic state. In addition, non-coding RNAs play important roles in the epigenetic regulation of leukemia. By regulating the expression of non-coding RNAs, processes such as proliferation, differentiation and apoptosis of leukemia cells can be affected. In summary, epigenetic therapy for leukemia is a novel therapeutic approach with potential. By intervening against the epigenetic abnormalities of leukemia cells, it can inhibit their malignant proliferation and induce differentiation or apoptosis, thus providing better therapeutic options for leukemia patients. In the future, with the in-depth study of the epigenetic mechanism of leukemia and the development of novel epigenetic drugs, epigenetic therapy is expected to become one of the important means of leukemia treatment.