Immune responses to AAV-FVIII gene therapy
Mechanisms of Innate and Adaptive Immune Responses to AAV-FVIII Gene Transfer
Working to reduce immune reactions that shorten the benefits of AAV-based gene therapy 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-11325363 on NIH RePORTER |
What this research studies
This project looks at why liver-directed AAV-FVIII gene therapy can lose effectiveness over time and cause liver inflammation. The team studies how the body makes antibodies and T-cell responses against the AAV vector and the FVIII protein using laboratory experiments, animal models, and samples from clinical trials. They aim to identify mechanisms that drive loss of factor VIII expression and liver toxicity and to test strategies to prevent those immune reactions. Findings will be used to guide safer, more durable gene therapy approaches for people with hemophilia A.
Who could benefit from this research
Good fit: People with hemophilia A (factor VIII deficiency) who are considering, enrolled in, or providing samples for AAV-FVIII gene therapy research would be the ideal candidates for related clinical or sample-donation activities.
Not a fit: People without hemophilia A or those who are ineligible for liver-directed AAV therapies (for example because of pre-existing AAV antibodies or significant liver disease) are unlikely to directly benefit from this work.
Why it matters
Potential benefit: Could lead to safer, more durable AAV-FVIII gene therapies that reduce or eliminate the need for regular factor infusions in people with hemophilia A.
How similar studies have performed: Some AAV-FVIII clinical trials have restored factor VIII levels in the short term, but many have shown declining expression over years and immune-related complications remain a key challenge.
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.