Therapeutic targeting of transcription-epigenetic defects in SF3B1-mutant MDS
Official title: Therapeutic Targeting of Transcription-Driven Epigenetic Defects in SF3B1 Mutant Myelodysplastic syndromes (MDS)
Funded at Yale University · NIH-11332844 · Data updated 2026-11-12
A training-focused grant to help a clinician-scientist develop therapies for adults with a common bone marrow failure condition called SF3B1-mutant myelodysplastic syndrome (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 start | July 1, 2024 |
| Project end | May 31, 2029 |
| Funding year | 2026 |
| Award (this year) | $168,048 |
| NIH project number | 5K08DK140636-03 |
| Project ID | NIH-11332844 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with myelodysplastic syndrome, particularly those with SF3B1 mutations, who may benefit from future translational therapies.
✕Less likely to help
People without SF3B1-mutant MDS or those not a candidate for therapeutic development may not benefit.
What this research studies
This award supports Dr. Prajwal Boddu in a mentorship-driven program to translate basic discoveries about how SF3B1 mutations change RNA transcription and epigenetics into potential treatments for MDS. The research explores how altered transcription elongation and chromatin state contribute to disease and aims to identify targetable epigenetic changes. The plan combines hands-on laboratory work with career development activities to build independent clinical-scientist capabilities. If successful, the work could point to new strategies to improve blood cell formation in patients with SF3B1-mutant MDS.
Why it matters
Potential benefit: If successful, patients with SF3B1-mutant MDS could gain new therapeutic options that address the underlying epigenetic and transcriptional defects.
How similar studies have gone: Previous work has linked SF3B1 mutations to splicing defects and epigenetic changes, with emerging evidence that targeting these pathways can influence disease.
Where this research is happening
-
Yale University
New Haven, United States Active
Researchers
- Principal investigator: Boddu, Prajwal — Yale University
- Study coordinator: Boddu, Prajwal
Related research
Other NIH-funded projects on the same topics.
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Targeting genetic defects in a type of blood disorder called myelodysplastic syndrome.
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.
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Understanding how SF3B1 mutations affect blood cell development in myelodysplastic syndromes.
This study is looking at how changes in a specific gene (SF3B1) affect blood cell production in people with myelodysplastic syndromes (MDS), using advanced techniques to understand these changes at the single-cell level, which could help improve treatments for the condition.
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Interferon-γ plus donor immune cells to fight AML/MDS after partial donor transplant
This project tests a therapy that adds a signaling protein, IFN-γ, with donor immune cells to help treat relapsed AML/MDS after a haploidentical stem cell transplant.
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Whole-genome sequencing to refine risk in myelodysplastic syndromes (MDS) for older adults
This project uses clinical whole-genome sequencing to improve genetic profiling and better predict how MDS will progress in older patients.
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Targeting gasdermin D to treat myelodysplastic syndromes in people with bone marrow inflammation
A research project aims to find new ways to treat myelodysplastic syndromes by blocking a protein (gasdermin D) involved in harmful bone marrow inflammation.
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Understanding why some MDS patients relapse after a stem cell transplant
A study of patients with myelodysplastic syndromes after stem cell transplant to learn the cellular changes and gene activity that lead to relapse, with the goal of guiding future treatments.
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.