Understanding what makes gene therapy last
Official title: Exploring genomic and cellular determinants of gene therapy durability
Funded at University of Arkansas at Fayetteville · NIH-11195007 · Data updated 2026-11-12
Researchers are looking at genetic and cellular reasons gene therapy can fade or cause changes, aiming to help people with genetic conditions like Duchenne muscular dystrophy.
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 | 2025 |
| Award (this year) | $366,731 |
| NIH project number | 5R35GM155433-02 |
| Project ID | NIH-11195007 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with inherited conditions targeted by gene therapy efforts—such as Duchenne muscular dystrophy—or patients willing to provide blood or tissue samples for research are most directly relevant.
✕Less likely to help
This basic lab-focused work is unlikely to offer direct treatment or immediate benefits to patients seeking therapy right now.
What this research studies
This project uses lab experiments in cells and animal models to track how gene-editing tools (like CRISPR) and viral delivery (AAV) behave over time. Scientists will measure immune responses, look for unintended DNA changes such as vector integration, and profile genomes, epigenomes, and RNA to understand long-term effects. Work will combine molecular analyses with tissue and behavioral studies in models relevant to conditions such as Duchenne muscular dystrophy. If you or a loved one are considering gene therapy, this work aims to uncover why treatments sometimes stop working or cause unexpected effects.
Why it matters
Potential benefit: Could help make gene therapies safer and longer-lasting for people with genetic diseases.
How similar studies have gone: Gene therapies have produced major successes in conditions like spinal muscular atrophy and inherited blindness, but immune reactions and long-term durability remain unresolved challenges.
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.