Making AAV gene therapy safer for hemophilia A using lab-grown human liver tissue
Using human liver tissue equivalents to optimize AAV-mediated GT and better define age-related clinical risks
Uses lab-grown human liver tissue to make AAV-based gene therapy safer and more predictable for children and adults with hemophilia A.
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 ID | NIH-11294261 on NIH RePORTER |
What this research studies
Researchers grow human liver tissue in the lab that mimics livers at different ages and expose those tissues to AAV-based gene therapy to see how liver cells respond. They monitor immune and inflammatory reactions, signs of liver toxicity, and whether the viral DNA integrates into the host genome in ways that could raise cancer risk. The team compares responses that resemble children versus adults and studies how pre-existing anti-AAV antibodies and ectopic factor VIII expression in liver cells affect safety.
Who could benefit from this research
Good fit: People with severe hemophilia A (including older children and adults) who are considering or eligible for AAV-based gene therapy are the most relevant candidates for these findings.
Not a fit: People with other bleeding disorders, those not receiving AAV-based treatments, or individuals with medical contraindications such as very high anti-AAV antibodies may not receive direct benefit from this project.
Why it matters
Potential benefit: If successful, this work could reduce unexpected liver damage and guide safer use of AAV gene therapy for people with hemophilia A, especially before treating children.
How similar studies have performed: AAV gene therapies have shown lasting benefits for some bleeding disorders and promising results in hemophilia trials, but unexpected liver toxicity in some hemophilia A patients means using human liver tissue models is a newer approach to improve safety.
Where this research is happening
Winston-Salem, United States
- 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
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 below.