Mutation-independent gene therapy for CEP290-linked retinal degeneration
Official title: Treatment of CEP290-associated retinal degeneration
Funded at University of Iowa · NIH-11450964 · Data updated 2026-11-12
A patient-facing study exploring new AAV-based gene therapy approaches to help children with CEP290-related vision loss.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Iowa NIH-funded |
| Lab location | 1 site (Iowa City, United States) |
| Project start | September 1, 2026 |
| Project end | August 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $427,625 |
| NIH project number | 1R21EY038431-01A1 |
| Project ID | NIH-11450964 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are children (0-21) with CEP290-LCA or related retinal degenerations who could benefit from future gene therapy trials.
✕Less likely to help
Children without CEP290-linked vision loss or without eligible CEP290 mutations may not benefit from this specific approach.
What this research studies
This project focuses on developing broad gene therapy vectors that can treat CEP290-related Leber congenital amaurosis across many mutations, aiming to restore CEP290 function in the retina. Researchers will test different multi-vector AAV strategies in lab settings and in mouse models to compare how well they reconstitute the CEP290 gene and protect vision-related cells. The work builds on prior lab results showing feasibility in producing full-length CEP290 proteins and evaluates which approach works best across models. If successful, this could lead toward a more universal treatment option for children affected by CEP290-LCA.
Why it matters
Potential benefit: If the therapy works, it could preserve or improve vision for children with CEP290-LCA by restoring CEP290 protein function.
How similar studies have gone: Previous studies showed promising in vitro and mouse-model CEP290 repair using multi-vector strategies, but human data are not yet available.
Where this research is happening
-
University of Iowa
Iowa City, United States Active
Researchers
- Principal investigator: Seo, Seongjin — University of Iowa
- Study coordinator: Seo, Seongjin
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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.