Investigating gene therapy for hemophilia A using liver-targeted AAV vectors
Defining the therapeutic efficacy, tolerogenic potential, and genotoxicity of liver-targeted AAV gene therapy for hemophilia A
This study is looking at a new gene therapy for hemophilia A that uses a special virus to help the liver produce the missing clotting factor, and it’s testing different ways to give this treatment safely to babies before they are born or shortly after.
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-10889145 on NIH RePORTER |
What this research studies
This research focuses on developing a gene therapy approach for hemophilia A that targets the liver using adeno-associated virus (AAV) vectors. It aims to evaluate the effectiveness of this therapy, its potential to induce tolerance in the immune system, and any risks of genetic damage. The study will explore both neonatal and in utero gene therapy methods to address challenges such as pre-existing immunity to AAV and the risk of developing inhibitors to clotting factors. By using animal models, the research seeks to identify optimal conditions for safe and effective treatment.
Who could benefit from this research
Good fit: Ideal candidates for this research include newborns and adults diagnosed with hemophilia A who have not developed inhibitors to clotting factors.
Not a fit: Patients with severe pre-existing AAV immunity or those who have already developed inhibitors to clotting factors may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a long-lasting treatment option for hemophilia A, potentially reducing or eliminating the need for regular factor replacement therapy.
How similar studies have performed: Previous research has shown promise in gene therapy approaches for hemophilia, but this specific method using liver-targeted AAV vectors is still being explored and is considered 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.