How the immune system reacts to muscle gene therapy using AAV
Mechanism of immune response to muscle-directed AAV gene transfer
They are figuring out how people's immune systems respond when AAV-based gene therapy is given to muscles, especially for conditions like Duchenne muscular dystrophy.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Indiana University Indianapolis NIH-funded |
| Lab location | 1 site (Indianapolis, United States) |
| Project ID | NIH-11325270 on NIH RePORTER |
What this research studies
This project looks at how your immune system reacts when AAV gene therapy is given to muscles for conditions like Duchenne muscular dystrophy. Researchers will examine immune cells, antibodies, complement activation, and T cell responses using lab experiments and animal models that mimic human disease, including dogs with DMD. They will study why neutralizing antibodies form, why re-dosing often fails, and immune responses to gene-editing tools such as Cas9. The team plans to use these results to guide safer and more durable muscle-directed gene therapies.
Who could benefit from this research
Good fit: People with Duchenne muscular dystrophy or other severe muscle-wasting conditions who are considering, have received, or could donate samples for AAV-based gene therapies are the most relevant candidates.
Not a fit: People without muscle disease or those seeking immediate treatment are unlikely to gain direct clinical benefit from this mainly preclinical/translational research.
Why it matters
Potential benefit: If successful, this work could make AAV muscle gene therapies safer and longer-lasting and help enable repeat dosing when needed.
How similar studies have performed: Prior AAV gene-delivery efforts have achieved therapeutic gene expression in some cases, but immune responses such as neutralizing antibodies, T cell reactions, and complement activation have limited effectiveness and re-dosing, so this area is promising but still faces important challenges.
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.