Engineered tissue arrays to reduce immune reactions in dystrophin gene therapy
Official title: Engineered tissue arrays to streamline deimmunized DMD gene therapy vectors
Funded at University of Washington · NIH-11596025 · Data updated 2026-11-12
A small, patient-focused grant aims to lower immune responses to dystrophin gene therapy by designing tissue models that help make the therapy less likely to trigger T-cell reactions.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Washington NIH-funded |
| Lab location | 1 site (Seattle, United States) |
| Project start | September 10, 2023 |
| Project end | August 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $450,758 |
| NIH project number | 4R33NS133080-03 |
| Project ID | NIH-11596025 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with Duchenne muscular dystrophy who would be eligible for dystrophin gene therapy approaches and who could contribute samples or clinical data for immunogenicity studies.
✕Less likely to help
Patients with dystrophin variants not impacted by immune responses or those who are not candidates for gene therapy may see no direct benefit.
What this research studies
This grant explores how changing specific regions of the dystrophin protein can prevent immune system activation, which could help more patients receive effective gene therapy for Duchenne muscular dystrophy. Researchers will use engineered tissue arrays to study how different amino acid changes affect immune recognition by antigen-presenting cells and T cells. The goal is to identify modifications that reduce T-cell responses without compromising therapeutic function, using human-relevant models. If successful, the approach could lead to safer, longer-lasting dystrophin therapies for patients with DMD.
Why it matters
Potential benefit: If successful, patients could benefit from dystrophin gene therapies that are less likely to provoke harmful immune reactions, improving durability and effectiveness.
How similar studies have gone: Preclinical studies have shown immune responses to dystrophin epitopes in some patients, making this a promising but still emerging strategy.
Where this research is happening
-
University of Washington
Seattle, United States Active
Researchers
- Principal investigator: Odom, Guy Leary — University of Washington
- Study coordinator: Odom, Guy Leary
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.