Lowering PUM1 to raise fetal hemoglobin in adults
Official title: Investigating PUM1 mediated post-transcriptional regulation of human hemoglobin switching and erythropoiesis
Funded at Cleveland State University · NIH-11323526 · Data updated 2026-11-12
Seeing if reducing a protein called PUM1 can raise protective fetal hemoglobin in adults with sickle cell disease or beta‑thalassemia.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cleveland State University NIH-funded |
| Lab location | 1 site (Cleveland, United States) |
| Project start | April 1, 2023 |
| Project end | March 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $401,908 |
| NIH project number | 5R01DK134936-04 |
| Project ID | NIH-11323526 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with sickle cell disease or beta‑thalassemia, or people willing to donate blood samples for research on HbF, would be the best matches for this work.
✕Less likely to help
People without sickle cell disease or β‑thalassemia, and children under 21, are unlikely to get direct benefit from this project.
What this research studies
The team studies a protein (PUM1) that helps control how red blood cells switch from fetal to adult hemoglobin. In lab work using human red blood cell precursors and genetic knockdown methods, they measure whether lowering PUM1 increases fetal γ‑globin and overall HbF levels without harming red blood cell development. They examine how PUM1 binds γ‑globin mRNA and affects its stability and translation. Results will guide whether targeting PUM1 could become a new way to boost HbF for people with sickle cell disease or β‑thalassemia.
Why it matters
Potential benefit: If successful, targeting PUM1 could increase fetal hemoglobin and lessen symptoms in people with sickle cell disease or β‑thalassemia.
How similar studies have gone: Existing treatments like hydroxyurea and newer gene‑editing approaches that raise fetal hemoglobin have helped patients, but targeting PUM1 is a novel, less-tested laboratory strategy with encouraging early data.
Where this research is happening
-
Cleveland State University
Cleveland, United States Active
Researchers
- Principal investigator: Gnanapragasam, Merlin Nithya — Cleveland State University
- Study coordinator: Gnanapragasam, Merlin Nithya
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.