Treg-based tolerance to dystrophin immune responses in Duchenne muscular dystrophy
Official title: Treg-mediated tolerance of dystrophin immunity
Funded at University of California-Irvine · NIH-11629772 · Data updated 2026-11-12
A new grant aims to help people with Duchenne muscular dystrophy by teaching the immune system to tolerate the dystrophin treatment delivered by gene therapy.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California-Irvine NIH-funded |
| Lab location | 1 site (Irvine, United States) |
| Project start | September 23, 2026 |
| Project end | August 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $549,500 |
| NIH project number | 1R56NS147542-01 |
| Project ID | NIH-11629772 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with Duchenne muscular dystrophy who are candidates for AAV-microdystrophin gene therapy.
✕Less likely to help
If a patient’s immune profile does not respond to Treg-based strategies, they may not experience improved tolerability.
What this research studies
Researchers will use a mouse model of Duchenne to study how regulatory T cells (Tregs) can quiet immune responses to dystrophin delivered by AAV gene therapy. They will test whether Tregs suppress dystrophin-specific T cells through an IL-10–dependent pathway and create Treg-enhancing immunocytokines to target the therapy to muscles. The work combines immunology and gene therapy approaches to improve safety and effectiveness for patients who could benefit from microdystrophin treatments. If successful, this could reduce immune-related adverse events and expand access to gene therapies for DMD.
Why it matters
Potential benefit: If successful, the project could make dystrophin gene therapies safer and usable for more patients with Duchenne muscular dystrophy.
How similar studies have gone: Prior research shows immune responses can affect dystrophin therapies; this study explores a targeted Treg approach, which is promising but still early.
Where this research is happening
-
University of California-Irvine
Irvine, United States Active
Researchers
- Principal investigator: Villalta, Sergio Armando — University of California-Irvine
- Study coordinator: Villalta, Sergio Armando
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.