Creating new mouse models to understand and treat severe alpha-thalassemia.
Official title: Development and validation of novel mouse models and expression vectors for characterizing severe alpha-thalassemia pathophysiology and evaluating gene therapy approaches.
Funded at Children's Hosp of Philadelphia · NIH-11143790 · Data updated 2026-11-12
This study is creating new mouse models to help us learn more about severe alpha-thalassemia, a condition that affects red blood cells, and to explore possible gene therapy treatments that could help people with this disease.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | June 1, 2023 |
| Project end | May 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $607,388 |
| NIH project number | 5R01DK133475-03 |
| Project ID | NIH-11143790 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with severe alpha-thalassemia who may benefit from innovative treatment approaches.
✕Less likely to help
Patients with mild forms of alpha-thalassemia or those who do not have the condition may not receive any benefit from this research.
What this research studies
This research focuses on developing and validating new mouse models to better understand severe alpha-thalassemia, a condition caused by insufficient production of the alpha-globin protein. By studying these models, researchers aim to explore the mechanisms behind red blood cell synthesis and how it is affected by excess beta-globin chains. The project also involves evaluating gene therapy approaches that could potentially lead to new treatments for patients suffering from this disease. The ultimate goal is to improve the understanding of alpha-thalassemia and identify effective therapies that could benefit patients.
Why it matters
Potential benefit: If successful, this research could lead to new treatment options for patients with severe alpha-thalassemia, potentially reducing their reliance on blood transfusions.
How similar studies have gone: While there has been significant research on alpha-thalassemia, the development of novel mouse models for this specific condition is a relatively new and untested approach.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Rivella, Stefano — Children's Hosp of Philadelphia
- Study coordinator: Rivella, Stefano
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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.