Investigating gene therapy for hemophilia A using human liver cells
Human Hepatocyte and Discovery Core
This study is looking at ways to make gene therapy for hemophilia A better by using human liver cells to see how we can improve the delivery of a key clotting factor, and it's designed for people with hemophilia A who want to learn about new treatment options.
Quick facts
| Grant type | P01 program project |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Indiana University Indianapolis NIH-funded |
| Lab location | 1 site (Indianapolis, United States) |
| Project ID | NIH-11023039 on NIH RePORTER |
What this research studies
This research focuses on improving gene therapy for hemophilia A by using human liver cells to understand how adeno-associated viral (AAV) gene transfer of clotting factor FVIII can be made more effective. The study employs three different models: primary human hepatocyte cultures for laboratory studies, chimeric mice with human liver cells for in vivo testing, and mice with both human liver and immune cells to explore immune responses. By examining these interactions, the research aims to uncover the reasons behind the decline in FVIII expression and potential safety issues associated with current therapies.
Who could benefit from this research
Good fit: Ideal candidates for this research are males diagnosed with hemophilia A who may benefit from advanced gene therapy approaches.
Not a fit: Patients with other types of hemophilia or those who do not have a genetic basis for their condition may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective and safer gene therapies for patients with hemophilia A.
How similar studies have performed: Previous research has shown promise in using AAV gene therapy for hemophilia, but this approach aims to address specific challenges that have not yet been fully resolved.
Where this research is happening
Indianapolis, United States
- Indiana University Indianapolis — Indianapolis, United States (Active)
Researchers
- Principal investigator: De Jong, Ype Peter — Indiana University Indianapolis
- Study coordinator: De Jong, Ype Peter
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.