Understanding how mutations in a protein complex affect blood cell formation in certain blood cancers.
Official title: Molecular mechanisms of splicing dependency in cohesin-mutant myelodysplasia
Funded at Dana-Farber Cancer Inst · NIH-10993594 · Data updated 2026-11-12
This study is looking at how changes in a group of proteins important for blood cell function might cause problems in patients with myelodysplastic syndromes (MDS), and it hopes to find new ways to help treat this condition.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Dana-Farber Cancer Inst NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | December 1, 2023 |
| Project end | November 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $400,500 |
| NIH project number | 5R01HL171973-02 |
| Project ID | NIH-10993594 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are patients diagnosed with myelodysplastic syndromes who have mutations in the cohesin complex.
✕Less likely to help
Patients without cohesin mutations or those with other types of blood disorders may not benefit from this research.
What this research studies
This research investigates the role of mutations in the cohesin complex, which is crucial for the proper functioning of blood cells, particularly in myelodysplastic syndromes (MDS). By using genetically engineered models, the study aims to explore how these mutations disrupt normal splicing processes, which are essential for cell survival. The researchers will examine the interactions between cohesin proteins and splicing factors to identify potential therapeutic targets. The goal is to enhance our understanding of the disease and develop new treatment strategies for patients with cohesin-mutant MDS.
Why it matters
Potential benefit: If successful, this research could lead to new targeted therapies for patients with specific mutations in their blood cancer, improving treatment outcomes.
How similar studies have gone: Previous research has shown promising results in targeting genetic mutations in blood cancers, suggesting that this approach could be effective.
Where this research is happening
-
Dana-Farber Cancer Inst
Boston, United States Active
Researchers
- Principal investigator: Tothova, Zuzana — Dana-Farber Cancer Inst
- Study coordinator: Tothova, Zuzana
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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.