Targeting genetic defects in a type of blood disorder called myelodysplastic syndrome.
Therapeutic Targeting of Transcription-Driven Epigenetic Defects in SF3B1 Mutant Myelodysplastic syndromes (MDS)
This study is looking at how changes in the SF3B1 gene affect myelodysplastic syndromes (MDS), a condition that can cause problems with blood cell production, to help find new treatment options that could make life easier for patients with MDS.
Quick facts
| Grant type | Career grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Yale University NIH-funded |
| Lab location | 1 site (New Haven, United States) |
| Project ID | NIH-11124840 on NIH RePORTER |
What this research studies
This research investigates how mutations in the SF3B1 gene contribute to myelodysplastic syndromes (MDS), a condition that leads to bone marrow failure. The study focuses on understanding the role of these mutations in disrupting RNA transcription and how this affects blood cell formation. By exploring the molecular mechanisms involved, the research aims to identify potential therapeutic targets that could improve treatment options for patients with MDS. Patients may benefit from new therapies developed based on the findings of this research.
Who could benefit from this research
Good fit: Ideal candidates for this research are adults diagnosed with myelodysplastic syndromes, particularly those with SF3B1 mutations.
Not a fit: Patients without myelodysplastic syndromes or those with different genetic mutations may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that specifically target the underlying genetic defects in myelodysplastic syndromes.
How similar studies have performed: Previous research has shown promise in targeting genetic defects in blood disorders, suggesting that this approach could be effective.
Where this research is happening
New Haven, United States
- Yale University — New Haven, United States (Active)
Researchers
- Principal investigator: Boddu, Prajwal — Yale University
- Study coordinator: Boddu, Prajwal
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.