Abstract

Acute myeloid leukemia (AML) is a form of blood cancer that is characterized by the rapid growth of abnormal myeloid cells. Although the general therapeutic strategy in patients with AML has not changed substantially in more than 30 years, remarkable progress has been achieved in understanding the pathogenesis of AML. Genome-wide analyses have revealed genetic mutations and epigenetic dysregulations that are present in AML cells. Studies of leukemia stem cells have clarified their complex properties and functions in the development of AML, and have also led to the recent identification of pre-leukemic hematopoietic stem cells that undergo clonal evolution in healthy people. Translation of these new findings into the clinical setting is just beginning. This article focuses on recent advances in basic research on the molecular pathogenesis of AML. New strategies under investigation, including epigenetic therapies and immunotherapies, to provide better therapeutic options for AML patients, are also summarized.

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