Nuclear transcriptional condensates as drivers and therapeutic targets in NPM1 -mutated AML
Abstract
ABSTRACT: NPM 1-mutated acute myeloid leukemia (AML) is driven by aberrant HOX/MEIS1 expression, whose mechanistic basis remained unresolved for years. Recent paradigm-shifting studies show that mutant NPM1 organizes phase-separated nuclear condensates that concentrate transcriptional regulators at active chromatin, directly sustaining the pathogenic HOX/MEIS1 transcriptional program. This framework explains the activity of menin-KMT2A inhibitors, recently approved by the US Food and Drug Administration, in this AML subtype and positions disruption of these assemblies as a precision strategy to eliminate oncogenic transcription.
Fields of Study & Subjects
Acute Myeloid Leukemia Research
Retinoids in leukemia and cellular processes
Protein Degradation and Inhibitors
Publication details
- Journal / source
- Blood
- Publisher
- American Society of Hematology
- Volume
- 147
- Issue
- 20
- Pages
- 2291–2297
- Publication date
- 8 April 2026
- Language
- EN