Creating a new model to understand Usher syndrome.
Generation of a new model for Usher syndrome.
['FUNDING_R21'] · UPSTATE MEDICAL UNIVERSITY · NIH-11012814
This study is creating a new animal model to better understand how Usher syndrome affects vision and hearing, so researchers can find ways to help people with this condition keep or regain their sight.
Quick facts
| Phase | ['FUNDING_R21'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UPSTATE MEDICAL UNIVERSITY (nih funded) |
| Locations | 1 site (SYRACUSE, UNITED STATES) |
| Trial ID | NIH-11012814 on ClinicalTrials.gov |
What this research studies
This research focuses on developing a new animal model that mimics the severe retinal degeneration seen in patients with Usher syndrome, a condition that causes both blindness and hearing loss. By studying how mutations in the USH2A gene affect photoreceptor health, researchers aim to uncover the underlying mechanisms of this disease. The model will help in testing potential therapies aimed at preserving or restoring vision in affected individuals. Patients may benefit from insights gained through this research, which could lead to effective treatments in the future.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Usher syndrome type II or related conditions that involve retinal degeneration and hearing loss.
Not a fit: Patients with Usher syndrome type I or those without genetic mutations in the USH2A gene may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that preserve or restore vision for patients with Usher syndrome.
How similar studies have performed: Other research has successfully developed animal models for similar genetic conditions, providing a foundation for this approach.
Where this research is happening
SYRACUSE, UNITED STATES
- UPSTATE MEDICAL UNIVERSITY — SYRACUSE, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: HU, HUAIYU — UPSTATE MEDICAL UNIVERSITY
- Study coordinator: HU, HUAIYU
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.