New gene delivery methods for treating vision loss in Usher Syndrome Type 2A
Official title: Admin Supplement: Non-viral gene delivery platforms for the treatment of Usher Syndrome Type 2A
Funded at University of Houston · NIH-11213861 · Data updated 2026-11-12
This study is exploring new ways to use gene therapy to help people with Usher Syndrome Type 2A, a condition that causes vision loss and currently has no treatments, by testing safe methods to deliver helpful genes directly to the eye, with the hope of restoring vision in the future.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Houston NIH-funded |
| Lab location | 1 site (Houston, United States) |
| Project start | January 1, 2023 |
| Project end | December 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $79,344 |
| NIH project number | 3R01EY034671-03S1 |
| Project ID | NIH-11213861 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Usher Syndrome Type 2A who are experiencing vision loss.
✕Less likely to help
Patients with other forms of Usher Syndrome or those without genetic mutations related to USH2A may not benefit from this research.
What this research studies
This research focuses on developing innovative gene therapy techniques to treat vision loss caused by Usher Syndrome Type 2A, a condition with no current treatments. The approach utilizes DNA nanoparticles and hyaluronic acid nanospheres to deliver large therapeutic genes directly to the eye, aiming to restore vision. The research involves testing these delivery systems in animal models to ensure safety and effectiveness before considering human applications. Patients may benefit from advancements in gene therapy that could potentially halt or reverse vision loss associated with this genetic disorder.
Why it matters
Potential benefit: If successful, this research could lead to effective treatments for vision loss in patients with Usher Syndrome Type 2A.
How similar studies have gone: Previous research has shown promise in using similar gene delivery methods for other genetic disorders, indicating potential for success in this novel application.
Where this research is happening
-
University of Houston
Houston, United States Active
Researchers
- Principal investigator: Naash, Muna I. — University of Houston
- Study coordinator: Naash, Muna I.
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.