Understanding how gene therapy works and lasts in the body
Official title: Exploring genomic and cellular determinants of gene therapy durability
Funded at University of Arkansas at Fayetteville · NIH-10941793 · Data updated 2026-11-12
This study is looking at how the immune system affects gene therapy treatments, aiming to make them safer and more effective for people with conditions like spinal muscular atrophy and inherited blindness, so patients can enjoy longer-lasting benefits from these therapies.
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 | 2024 |
| Award (this year) | $367,059 |
| NIH project number | 1R35GM155433-01 |
| Project ID | NIH-10941793 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for participation or benefit include individuals with genetic disorders such as spinal muscular atrophy or inherited blindness.
✕Less likely to help
Patients with conditions not related to genetic disorders or those who do not qualify for gene therapy may not receive benefits from this research.
What this research studies
This research investigates the factors that influence the effectiveness and longevity of gene therapy treatments. By examining how the immune system responds to these therapies and the potential for unintended genetic changes, the study aims to improve the safety and success of gene therapies. Patients may benefit from insights into how to enhance the durability of these treatments, particularly for conditions like spinal muscular atrophy and inherited blindness. The research employs advanced gene-editing tools and delivery methods to explore these critical questions.
Why it matters
Potential benefit: If successful, this research could lead to more effective and lasting gene therapies for various genetic disorders.
How similar studies have gone: Previous research has shown promising results in gene therapy, but this study aims to address specific unknowns that could enhance treatment durability.
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.