Optimizing gene therapy for bleeding disorders using liver tissue models
Official title: Using human liver tissue equivalents to optimize AAV-mediated GT and better define age-related clinical risks
Funded at Wake Forest University Health Sciences · NIH-11015910 · Data updated 2026-11-12
This study is looking at how to make gene therapy safer for kids with hemophilia A by using special lab-made liver tissues to better understand how the treatment works and any risks it might have.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Wake Forest University Health Sciences NIH-funded |
| Lab location | 1 site (Winston-Salem, United States) |
| Project start | February 15, 2023 |
| Project end | January 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $383,800 |
| NIH project number | 5R01HL166462-03 |
| Project ID | NIH-11015910 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include children and adults with hemophilia A who are considering gene therapy options.
✕Less likely to help
Patients with bleeding disorders who are not candidates for gene therapy or those with contraindications to AAV vectors may not benefit from this research.
What this research studies
This research investigates the use of human liver tissue equivalents to improve gene therapy techniques that utilize adeno-associated viral (AAV) vectors for treating bleeding disorders, particularly hemophilia A. The study aims to understand the unexpected risks associated with AAV-mediated gene therapy, especially in children, by examining immune responses and potential liver toxicity. By analyzing how AAV interacts with liver cells and the implications of ectopic factor VIII expression, the research seeks to predict and mitigate risks for patients undergoing this treatment. The approach combines advanced tissue models with clinical insights to enhance the safety and efficacy of gene therapy.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective gene therapy treatments for patients with bleeding disorders.
How similar studies have gone: Previous research has shown promise in using AAV vectors for gene therapy, but this specific approach to understanding age-related risks is novel.
Where this research is happening
-
Wake Forest University Health Sciences
Winston-Salem, United States Active
Researchers
- Principal investigator: Almeida-Porada, Graca Duarte — Wake Forest University Health Sciences
- Study coordinator: Almeida-Porada, Graca Duarte
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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.