Understanding how gene therapy works over time in Duchenne-related disease
Official title: Exploring genomic and cellular determinants of gene therapy durability
Funded at University of Arkansas at Fayetteville · NIH-11396366 · Data updated 2026-11-12
Aiming to learn what affects how long gene therapy lasts and how the body reacts to it in Aran-Duchenne disease.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Arkansas at Fayetteville NIH-funded |
| Lab location | 1 site (Fayetteville, United States) |
| Project start | September 20, 2024 |
| Project end | August 31, 2029 |
| Funding year | 2026 |
| Award (this year) | $366,317 |
| NIH project number | 5R35GM155433-03 |
| Project ID | NIH-11396366 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with Aran-Duchenne disease or related conditions who may be candidates for future gene therapy trials.
✕Less likely to help
Participants in early safety-only studies or those not exposed to gene therapies may not benefit.
What this research studies
This grant explores how genomic and cellular changes influence how durable gene therapy is, using advanced gene-editing tools and delivery methods. Researchers will study immune responses, vector integration, and long-term genomic effects to understand why some therapies may lose their benefit over time. The work involves animal models and analyses of molecular data to map outcomes after gene therapy. Results could guide safer, longer-lasting treatments for patients with Aran-Duchenne disease.
Why it matters
Potential benefit: Could lead to longer-lasting and safer gene therapies for Aran-Duchenne disease.
How similar studies have gone: There is prior evidence that gene therapy can be durable in some models, but durability and immune responses remain variable and warrant further study.
Where this research is happening
-
University of Arkansas at Fayetteville
Fayetteville, United States Active
Researchers
- Principal investigator: Nelson, Christopher — University of Arkansas at Fayetteville
- Study coordinator: Nelson, Christopher
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.