Improving gene therapy for hemophilia using modified AAV3 vectors.
Official title: Capsid- and genome-modified AAV3 vectors for hemophilia gene therapy.
Funded at University of Florida · NIH-11024765 · Data updated 2026-11-12
This study is working on improving gene therapy for hemophilia A and B by creating better delivery systems that help get the right genes into liver cells, which could lead to safer and more effective treatments for people with these bleeding disorders.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Florida NIH-funded |
| Lab location | 1 site (Gainesville, United States) |
| Project start | December 15, 2024 |
| Project end | November 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $704,635 |
| NIH project number | 1R01HL177230-01 |
| Project ID | NIH-11024765 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with hemophilia A or B who are seeking innovative treatment options.
✕Less likely to help
Patients with other bleeding disorders not related to hemophilia may not receive benefits from this research.
What this research studies
This research focuses on enhancing gene therapy for hemophilia A and B by developing modified adeno-associated virus (AAV3) vectors. The approach involves creating capsid- and genome-modified AAV3 vectors that are more efficient in delivering therapeutic genes to liver cells, which are crucial for producing clotting factors. By optimizing these vectors, the research aims to improve transgene expression and reduce the need for high doses and immune suppression. Patients may benefit from a more effective and safer gene therapy option for their bleeding disorders.
Why it matters
Potential benefit: If successful, this research could provide a more effective and safer gene therapy for patients with hemophilia, potentially reducing bleeding episodes and improving quality of life.
How similar studies have gone: Previous research has shown success with AAV vectors in gene therapy for hemophilia, but this approach with AAV3 is novel and aims to improve upon existing methods.
Where this research is happening
-
University of Florida
Gainesville, United States Active
Researchers
- Principal investigator: Srivastava, Arun — University of Florida
- Study coordinator: Srivastava, Arun
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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.