Nucleoporin changes linked to aging and progression from MDS to AML
Molecular Mechanisms of MDS pathogenesis with aging
Finding out whether lower levels of nucleoporin proteins in blood stem cells help explain why myelodysplastic syndromes (MDS) worsen into acute myeloid leukemia (AML) in older adults.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-11374450 on NIH RePORTER |
What this research studies
This project looks at how changes in a family of proteins called nucleoporins (NUPs) relate to MDS and its progression to AML, with attention to aging. Researchers will compare results from three mouse MDS models, patient blood and bone marrow cells (including paired MDS-to-AML samples), and patient-derived iPSC models to study the effects of lowering NUPs. They will examine links between NUP expression, DNMT3A-related clonal hematopoiesis, and a transcriptional signature tied to disease transformation. The goal is to find molecular pathways in disease-initiating stem cells that could be targeted to prevent or reverse progression to leukemia.
Who could benefit from this research
Good fit: People with myelodysplastic syndromes (especially older adults), those with DNMT3A-associated clonal hematopoiesis, or patients whose MDS shows signs of transforming toward AML would be most relevant.
Not a fit: People without MDS or with unrelated blood disorders are unlikely to receive direct benefit from this project.
Why it matters
Potential benefit: If successful, this work could identify new molecular targets to stop MDS from progressing to AML and help develop treatments that eliminate the disease-initiating stem cells.
How similar studies have performed: Preclinical studies and patient-sample analyses have suggested links between NUP expression changes and disease progression, but converting these findings into human therapies remains largely untested.
Where this research is happening
New York, United States
- Columbia University Health Sciences — New York, United States (Active)
Researchers
- Principal investigator: Kousteni, Stavroula — Columbia University Health Sciences
- Study coordinator: Kousteni, Stavroula
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.