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Accurate Medicine: Indirect Targeting of NPM1-Mutated AML
Christopher S Hourigan1, Peter D Aplan2
1Myeloid Malignancies Section, Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland. aplanp@mail.nih.gov christopher.hourigan@nih.gov.
Abstract:
Acute myeloid leukemia (AML) is now recognized to be an imprecise term that refers to a range of myeloid malignancies that have different genetical etiologies, clinical characteristics, and therapeutic sensitivities. Targeting the MLL1 and DOT1L histone modification complexes, both alone and in combination, showed activity against AML driven by a mutant NPM1 protein in several preclinical models and may represent a new treatment direction for this devastating disease. Cancer Discov; 6(10); 1087-9 ©2016 AACR.See related article by Kühn and colleagues p. 1166.

