Understanding how U2AF1 mutations cause blood disorders
Official title: Molecular Basis for Myelodysplasia Induced by U2AF1 Mutations
Funded at University of Virginia · NIH-11097324 · Data updated 2026-11-12
This study is looking at how changes in a specific gene called U2AF1 affect blood cell production in people with myelodysplastic syndromes (MDS), with the hope of finding new ways to improve treatments for patients like you.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Virginia NIH-funded |
| Lab location | 1 site (Charlottesville, United States) |
| Project start | June 15, 2023 |
| Project end | May 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $581,381 |
| NIH project number | 5R01HL168611-03 |
| Project ID | NIH-11097324 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are patients diagnosed with myelodysplastic syndromes or those with mutations in the U2AF1 gene.
✕Less likely to help
Patients with blood disorders not related to myelodysplastic syndromes or U2AF1 mutations may not benefit from this research.
What this research studies
This research investigates the molecular mechanisms behind myelodysplastic syndromes (MDS), which are blood disorders that can lead to acute myeloid leukemia. The study focuses on mutations in the U2AF1 gene, which are commonly found in MDS patients. By using a specially designed mouse model, researchers aim to uncover how these mutations affect blood cell formation and contribute to disease progression. The goal is to identify new therapeutic targets that could lead to better treatments for patients suffering from MDS.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that improve outcomes for patients with myelodysplastic syndromes and related blood disorders.
How similar studies have gone: Previous research has shown that targeting RNA splicing factors can be effective in treating blood disorders, suggesting that this approach may yield promising results.
Where this research is happening
-
University of Virginia
Charlottesville, United States Active
Researchers
- Principal investigator: Mohi, Golam — University of Virginia
- Study coordinator: Mohi, Golam
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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.