PARP1 and targeted treatment for myelodysplastic syndromes
Official title: Implications of PARP1 in myelodysplastic syndromes and targeted therapy
Funded at University of Minnesota · NIH-11325793 · Data updated 2026-11-12
This work tests whether blocking a protein called PARP1 can help people with myelodysplastic syndromes who have certain RNA splicing gene mutations.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Minnesota NIH-funded |
| Lab location | 1 site (Minneapolis, United States) |
| Project start | May 20, 2022 |
| Project end | April 30, 2027 |
| Funding year | 2026 |
| Award (this year) | $501,122 |
| NIH project number | 5R01HL163011-05 |
| Project ID | NIH-11325793 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are people with a diagnosis of myelodysplastic syndrome whose disease has mutations in RNA splicing genes such as U2AF1, SF3B1, SRSF2, or ZRSR2.
✕Less likely to help
Patients without these specific splicing mutations or whose MDS is driven by other mechanisms are less likely to benefit from PARP-targeted approaches.
What this research studies
You would be hearing about research that looks at how mutations in RNA splicing genes cause harmful RNA:DNA structures called R-loops that damage bone marrow cells. Scientists will study cells carrying these MDS-associated mutations and measure how PARP1 responds to R-loops and DNA damage. They will then test whether drugs that block PARP1 make those mutant cells more likely to die, using laboratory models and likely patient-derived samples. The goal is to find if existing PARP inhibitor drugs could be repurposed for people with specific genetic forms of MDS.
Why it matters
Potential benefit: If successful, this could point to a new targeted treatment option using PARP inhibitors for patients with MDS who have spliceosome mutations.
How similar studies have gone: PARP inhibitors are already effective in cancers with DNA repair defects (for example BRCA-mutant breast and ovarian cancers), and early lab studies suggest the idea may also work in MDS with splicing mutations, but clinical proof in MDS is still emerging.
Where this research is happening
-
University of Minnesota
Minneapolis, United States Active
Researchers
- Principal investigator: Nguyen, Dang Hai — University of Minnesota
- Study coordinator: Nguyen, Dang Hai
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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.