Investigating gene editing techniques for eye diseases
Viral Production and CRISPR Engineering
This study is working on new ways to use viruses to help fix genes that cause vision problems, like age-related macular degeneration, with the hope of finding better treatments for people who have trouble seeing.
Quick facts
| Grant type | P30 center grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Diego NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project ID | NIH-11092250 on NIH RePORTER |
What this research studies
This research focuses on developing advanced viral vectors to deliver gene editing tools, specifically targeting conditions like age-related macular degeneration. By utilizing adeno-associated viruses (AAV) and lentiviruses, the team aims to create conditions that either activate or inhibit specific genes involved in eye diseases. The approach includes using CRISPR technology to modify genes in rodent models, which could lead to new treatments for patients with vision impairments. The research also explores the potential of these vectors to express therapeutic genes directly.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with age-related macular degeneration or other inherited retinal degenerations.
Not a fit: Patients with non-genetic forms of vision impairment or those not affected by the targeted eye diseases may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to innovative gene therapies that improve or restore vision in patients suffering from age-related macular degeneration and other eye diseases.
How similar studies have performed: Previous research has shown promising results using similar gene editing approaches in other conditions, indicating potential for success in this area as well.
Where this research is happening
La Jolla, United States
- University of California, San Diego — La Jolla, United States (Active)
Researchers
- Principal investigator: Welsbie, Derek Stuart — University of California, San Diego
- Study coordinator: Welsbie, Derek Stuart
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.