Making gene therapy for hemophilia safer by improving immune control
Enhancing immune regulation in gene therapy for hemophilia
['FUNDING_R01'] · INDIANA UNIVERSITY INDIANAPOLIS · NIH-11243917
This project aims to reduce harmful immune reactions so gene therapy for people with hemophilia works better and lasts longer.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | INDIANA UNIVERSITY INDIANAPOLIS (nih funded) |
| Locations | 1 site (INDIANAPOLIS, UNITED STATES) |
| Trial ID | NIH-11243917 on ClinicalTrials.gov |
What this research studies
From a patient perspective, the team studies how AAV liver-directed gene therapy interacts with the immune system to either teach the body to tolerate the new clotting factor or trigger harmful responses. They use animal models and human-relevant systems to track regulatory T cells, CD8+ T cell activation, and signals from liver immune cells that influence those responses. The researchers are testing added immune-modulating approaches and exploring how conditions like fatty liver affect outcomes to find ways to prevent antibodies and other immune problems. Their methods combine gene transfer experiments, cellular and molecular immune studies, and translational models that reflect human biology.
Who could benefit from this research
Good fit: People with hemophilia A or B who are candidates for or interested in liver-directed AAV gene therapy, especially those with problematic antibodies to clotting factors, would be most directly relevant.
Not a fit: People without hemophilia or those who cannot receive AAV-based liver gene therapy (for example due to high pre-existing anti-AAV antibodies) are unlikely to benefit from this work.
Why it matters
Potential benefit: If successful, this work could lower immune-related risks and broaden who can safely benefit from AAV gene therapy for hemophilia.
How similar studies have performed: Liver-directed AAV gene therapies for hemophilia have shown promise and received regulatory approval, and animal studies plus early human reports suggest immune tolerance can occur, but immune responses remain a key challenge this work builds on.
Where this research is happening
INDIANAPOLIS, UNITED STATES
- INDIANA UNIVERSITY INDIANAPOLIS — INDIANAPOLIS, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: HERZOG, ROLAND W. — INDIANA UNIVERSITY INDIANAPOLIS
- Study coordinator: HERZOG, ROLAND W.
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.