Non-viral gene delivery for treating vision loss in Usher syndrome type 2A
Non-viral gene delivery platforms for the treatment of Usher Syndrome Type 2A.
['FUNDING_R01'] · UNIVERSITY OF HOUSTON · NIH-11248393
A new non-viral eye gene-delivery method is being developed to try to restore or protect vision in people with Usher syndrome type 2A.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF HOUSTON (nih funded) |
| Locations | 1 site (HOUSTON, UNITED STATES) |
| Trial ID | NIH-11248393 on ClinicalTrials.gov |
What this research studies
This project uses tiny DNA nanoparticles and hyaluronic acid nanospheres that can be injected into the eye to carry very large genes that do not fit into standard viral carriers. Researchers will test these carriers in lab models and in animals (mice and baboons) to see if they reach the light-sensing photoreceptor cells and safely restore function for the common USH2A c.2299delG mutation. The team will also use a small molecule called sulfotyrosine to help particles penetrate the retina and engineered DNA vectors to hold and express the large usherin gene. Positive results in these studies would support moving toward human clinical trials for people with USH2A.
Who could benefit from this research
Good fit: People with a confirmed genetic diagnosis of Usher syndrome type 2A, particularly those carrying the common c.2299delG mutation and who still have viable photoreceptors, would be the likely candidates for eventual trials.
Not a fit: People with vision loss from unrelated conditions, very advanced retinal degeneration with few remaining photoreceptors, or without a USH2A mutation are unlikely to benefit from this approach.
Why it matters
Potential benefit: If successful, this approach could preserve or restore vision for people with USH2A by delivering the large usherin gene without using viral vectors.
How similar studies have performed: Related DNA-nanoparticle and hyaluronic acid carrier methods have restored retinal structure or function in animal models, but delivering very large genes like USH2A to humans remains a novel and unproven step.
Where this research is happening
HOUSTON, UNITED STATES
- 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 below.