Developing gene therapy to treat alpha thalassemia
Official title: Developing gene therapy strategies to treat alpha thalassemia
Funded at University of California, San Francisco · NIH-11004158 · Data updated 2026-11-12
This study is exploring new gene therapy methods to help people with severe alpha thalassemia, a blood disorder that makes it hard for your body to produce healthy hemoglobin, by using cutting-edge techniques to fix the genes that cause the problem, offering hope for a better treatment than the current options like blood transfusions or stem cell transplants.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project start | January 1, 2022 |
| Project end | December 31, 2025 |
| Funding year | 2025 |
| Award (this year) | $627,518 |
| NIH project number | 5R01HL161291-04 |
| Project ID | NIH-11004158 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with severe forms of alpha thalassemia, particularly those requiring chronic transfusions or stem cell transplantation.
✕Less likely to help
Patients with mild forms of alpha thalassemia or those who do not require ongoing treatment may not benefit from this research.
What this research studies
This research focuses on creating innovative gene therapy strategies to address alpha thalassemia, a severe blood disorder caused by genetic mutations affecting hemoglobin production. The approach involves using advanced techniques like CRISPR and AAV-mediated genome editing to potentially replace faulty genes responsible for the disease. By adapting successful treatments from beta-thalassemia, the research aims to provide a new therapeutic option for patients suffering from the most severe forms of alpha thalassemia, which currently require frequent blood transfusions or stem cell transplants.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment that alleviates the need for regular blood transfusions and improves the quality of life for patients with alpha thalassemia.
How similar studies have gone: Previous research has shown success in using gene therapy for beta-thalassemia, suggesting a promising potential for similar approaches in treating alpha thalassemia.
Where this research is happening
-
University of California, San Francisco
San Francisco, United States Active
Researchers
- Principal investigator: Mackenzie, Tippi — University of California, San Francisco
- Study coordinator: Mackenzie, Tippi
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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.