New gene delivery methods to treat vision loss in Usher Syndrome Type 2A.
Non-viral gene delivery platforms for the treatment of Usher Syndrome Type 2A.
This study is exploring a new way to use tiny particles to deliver helpful genes directly into the eye to treat vision loss caused by Usher Syndrome Type 2A, with the hope of creating a safe and effective treatment for people affected by this condition.
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 ID | NIH-11012358 on NIH RePORTER |
What this research studies
This research focuses on developing innovative gene therapy techniques using DNA nanoparticles and hyaluronic acid nanospheres to deliver large therapeutic genes for treating vision loss associated with Usher Syndrome Type 2A. The approach aims to safely and effectively rescue vision by targeting the specific genetic mutation responsible for this condition. By utilizing advanced nanoformulation and a deep understanding of the visual and auditory systems, the research seeks to create a viable treatment option where none currently exists. The methodology includes intravitreal delivery, which allows for direct administration into the eye, enhancing the potential for successful gene expression.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Usher Syndrome Type 2A who are experiencing vision loss.
Not a fit: Patients with Usher Syndrome Type 2A who are not experiencing vision loss or those with other forms of Usher Syndrome may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for patients suffering from vision loss due to Usher Syndrome Type 2A.
How similar studies have performed: While gene therapy for other conditions has shown promise, this specific approach for Usher Syndrome Type 2A is novel and has not been extensively tested.
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.