Understanding immune responses to gene therapy for hemophilia
Mechanisms of Innate and Adaptive Immune Responses to AAV-FVIII Gene Transfer
This study is looking at how your immune system reacts to a new gene therapy for hemophilia, with the goal of making the treatment work better and last longer, so you can have safer and more effective options in the future.
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-11023070 on NIH RePORTER |
What this research studies
This research investigates how the immune system responds to gene therapy using adeno-associated viral (AAV) vectors to treat hemophilia, a bleeding disorder. It focuses on both innate and adaptive immune responses, aiming to understand the mechanisms that affect the stability and effectiveness of the treatment. By studying these immune responses, the research seeks to improve the durability of factor VIII expression, which is crucial for managing hemophilia. Patients may benefit from insights that could lead to safer and more effective gene therapy options.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with hemophilia A who are considering or currently participating in gene therapy trials.
Not a fit: Patients with hemophilia B or those who do not have a bleeding disorder may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective and long-lasting treatments for hemophilia, potentially reducing the need for frequent injections of coagulation factors.
How similar studies have performed: Previous research has shown promise in using AAV vectors for gene therapy in hemophilia, but challenges remain regarding the stability of factor VIII expression.
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.