MYELOID DISEASE: Edited by Martin S. TallmanDriver mutations in acute myeloid leukemiaKishtagari, Ashwina,b; Levine, Ross L.c,d; Viny, Aaron D.c,dAuthor Information aDepartment of Translational Hematology and Oncology Research bDepartment of Hematology and Oncology, Taussig Cancer Institute, Cleveland Clinic, Cleveland, Ohio cHuman Oncology and Pathogenesis Program dDepartment of Medicine, Leukemia Service, Memorial Sloan Kettering Cancer Center, New York, New York, USA Correspondence to Aaron D. Viny, Leukemia Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, 1275 York Avenue, Box 20, New York, NY 10065, USA. E-mail: [email protected] Current Opinion in Hematology: March 2020 - Volume 27 - Issue 2 - p 49-57 doi: 10.1097/MOH.0000000000000567 Buy Metrics Abstract Purpose of review The mutational landscape of acute myeloid leukemia (AML) has revised diagnostic, prognostic, and therapeutic schemata over the past decade. Recurrently mutated AML genes have functional consequences beyond typical oncogene-driven growth and loss of tumor suppresser function. Recent findings Large-scale genomic sequencing efforts have mapped the complexity of AML and trials of mutation-based targeted therapy has led to several FDA-approved drugs for mutant-specific AML. However, many recurrent mutations have been identified across a spectrum from clonal hematopoiesis to myelodysplasia to overt AML, such as effectors of DNA methylation, chromatin modifiers, and spliceosomal machinery. The functional effects of these mutations are the basis for substantial discovery. Summary Understanding the molecular and pathophysiologic functions of key genes that exert leukemogenic potential is essential towards translating these findings into better treatment for AML. Copyright © 2020 Wolters Kluwer Health, Inc. All rights reserved.