What triggers acute myeloid leukemia at the molecular level
Molecular Pathogenesis of Acute Myeloid Leukemia
Researchers are using patient leukemia samples, lab-grown stem cells, and genetic mouse models to find the molecular changes that start AML so new targeted treatments can be developed.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Washington University NIH-funded |
| Lab location | 1 site (Saint Louis, United States) |
| Project ID | NIH-11145211 on NIH RePORTER |
What this research studies
As a patient with AML, this project looks for the earliest molecular changes that let blood stem cells become leukemia. The team uses primary human AML samples, induced pluripotent stem cells, and genetically engineered mice to compare pre-leukemic and fully transformed cells. They apply DNA and RNA sequencing, whole-genome bisulfite sequencing, ATAC-seq, ChIP-seq/CUT&RUN, single-cell sequencing, and proteomics to create detailed molecular maps. The work focuses on common initiating events such as DNMT3A mutations and core binding factor fusions to guide future precision treatments.
Who could benefit from this research
Good fit: Ideal contributors would be people with AML, especially those with DNMT3A mutations or RUNX1-RUNX1T1/CBFB-MYH11 fusions, who are willing to provide diagnostic or research blood or bone marrow samples.
Not a fit: Patients seeking immediate clinical improvement or those without AML-related initiating mutations are unlikely to see direct benefit in the near term.
Why it matters
Potential benefit: If successful, this work could identify precise molecular targets that lead to more effective, personalized therapies for people with AML.
How similar studies have performed: Previous genomic and epigenomic studies have revealed mechanisms and candidate targets in AML, but turning those findings into proven precision treatments is still limited.
Where this research is happening
Saint Louis, United States
- Washington University — Saint Louis, United States (Active)
Researchers
- Principal investigator: Ley, Timothy J. — Washington University
- Study coordinator: Ley, Timothy J.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.