NPM1-mutated AML: targeting by disassembling.

摘要

In this issue of Blood, Balusu and collegues provide preclinical evidences that targeting nucleophosmin (NPM1) induces differentiation and death of acute myeloid leukemia (AML) cells harboring NPM1 mutations. These findings establish the rationale for novel treatment strategies in this large subgroup of AML. NPM1 is one of the most abundant proteins in the nucleolus. Despite its nucleolar localization, NPM1 physiologically shuttles constantly across various cell compartments (nucleolus, nucleoplasm, cytoplasm) and this traffic is critical for most of its functions, including regulation of ribosome biogenesis and control of centrosome duplication. NPM1 also interacts with the tumor suppressor p14~(ARF) and p53, and influences the cellular apoptotic response, although its exact role in this pathway still remains poorly understood.