Rau et al. treated primary cells from DNMT3A mutation-related AML patients with H3K79 methyltransferase Dot1L inhibitor EPZ5676; Dot1L is the key downstream gene overexpressed in hematopoietic stem cells, which regulates leukemia-related genes and promoted the development of disease in their Dnmt3a knockout murine, which significantly inhibited colony formation and induced terminal differentiation of the primary cells [38]. The gene discussed is DOT1L; the disease is leukemia.