Understanding how to safely increase fetal hemoglobin for treating blood disorders
Official title: Defining the mechanisms of hemoglobin switching and genotoxicities associated with its manipulation
Funded at Versiti Wisconsin, INC. · NIH-11195974 · Data updated 2026-11-12
This study is looking at how to safely boost fetal hemoglobin levels in people with blood disorders like beta-thalassemia, by exploring new ways to change certain cells in the body, so that patients can have better treatment options.
Quick facts
| Grant type | Career grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Versiti Wisconsin, INC. NIH-funded |
| Lab location | 1 site (Milwaukee, United States) |
| Project start | September 21, 2024 |
| Project end | November 30, 2026 |
| Funding year | 2025 |
| Award (this year) | $129,319 |
| NIH project number | 7K01DK132453-04 |
| Project ID | NIH-11195974 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adults with beta-hemoglobinopathies, such as beta-thalassemia, who may benefit from increased fetal hemoglobin levels.
✕Less likely to help
Patients with other types of blood disorders or those who do not have beta-hemoglobinopathies may not receive any benefit from this research.
What this research studies
This research investigates the mechanisms behind fetal hemoglobin production and the potential risks associated with genome editing techniques used to enhance its levels in patients with beta-hemoglobinopathies. The study focuses on manipulating hematopoietic stem and progenitor cells to induce fetal hemoglobin, while also examining the genetic changes that may occur as a result of this manipulation. By identifying key regulatory elements in the gamma-globin gene, the research aims to improve therapeutic strategies for patients affected by blood disorders. The findings could lead to safer and more effective treatments for conditions like beta-thalassemia.
Why it matters
Potential benefit: If successful, this research could provide a novel and safer therapeutic approach for patients with beta-hemoglobinopathies, potentially improving their quality of life.
How similar studies have gone: Other research has shown promise in using genome editing to increase fetal hemoglobin, but this specific approach and its safety profile are still being explored.
Where this research is happening
-
Versiti Wisconsin, INC.
Milwaukee, United States Active
Researchers
- Principal investigator: Doerfler, Phillip a — Versiti Wisconsin, INC.
- Study coordinator: Doerfler, Phillip a
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.