Optimizing gene therapy for bleeding disorders using liver tissue models
Using human liver tissue equivalents to optimize AAV-mediated GT and better define age-related clinical risks
['FUNDING_R01'] · WAKE FOREST UNIVERSITY HEALTH SCIENCES · NIH-11015910
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
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | WAKE FOREST UNIVERSITY HEALTH SCIENCES (nih funded) |
| Locations | 1 site (WINSTON-SALEM, UNITED STATES) |
| Trial ID | NIH-11015910 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research include children and adults with hemophilia A who are considering gene therapy options.
Not a fit: Patients with bleeding disorders who are not candidates for gene therapy or those with contraindications to AAV vectors may not benefit from this research.
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 performed: 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
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.