Human liver cell models to improve gene therapy for hemophilia A
Human Hepatocyte and Discovery Core
Using human liver cells and humanized mouse models to find why AAV gene therapy for hemophilia A can lose effect or harm the liver, with the aim of making treatments last longer and safer for people with hemophilia A.
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-11325356 on NIH RePORTER |
What this research studies
Researchers will use three human-based liver models to study AAV-delivered FVIII gene therapy from a patient's point of view. The team will grow primary human hepatocytes in lab dishes that keep liver cells working, use mice whose livers are largely repopulated with human hepatocytes for in-body testing, and use mice that also carry human immune cells to study immune reactions. They will look specifically at how the AAV vectors interact with human liver cells, how FVIII expression changes over time, and what causes liver injury or loss of benefit. The aim is to inform safer, longer-lasting gene therapy approaches for people living with hemophilia A.
Who could benefit from this research
Good fit: Ideal participants would be people with hemophilia A—particularly those interested in or considering AAV gene therapy—or individuals who can donate liver tissue or blood cells for research.
Not a fit: People with bleeding disorders not caused by factor VIII deficiency or those who are not candidates for liver-directed treatments are unlikely to benefit directly from this work.
Why it matters
Potential benefit: Could lead to AAV gene therapies for hemophilia A that work for longer and have fewer liver or immune-related side effects.
How similar studies have performed: Previous AAV gene therapy trials for hemophilia A have shown initial increases in FVIII but often declining levels and some liver toxicity, so this project builds on existing work to address those persistent problems.
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.