Eye-injection gene therapy for inherited retinal disease
Intravitreal gene therapy for inherited retinal disease
A gene therapy delivered by an injection into the eye aims to treat inherited forms of retinal degeneration, including in children.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California at Davis NIH-funded |
| Lab location | 1 site (Davis, United States) |
| Project ID | NIH-11115686 on NIH RePORTER |
What this research studies
This work develops a viral gene therapy given as an intravitreal (eye) injection using AAV vectors to reach and protect photoreceptor cells in inherited retinal diseases. Compared with current subretinal surgery, the goal is a less invasive delivery that can treat a larger area of the retina and avoid macular detachment and related surgical risks. The team will optimize AAV designs and delivery methods in laboratory and preclinical models and advance promising approaches toward early human testing. If successful, the approach could expand who can receive gene therapy and reduce the need for complex eye surgery.
Who could benefit from this research
Good fit: People (including children) with genetically confirmed single-gene inherited retinal degenerations who meet clinical and safety criteria for AAV-based therapy.
Not a fit: Those whose vision loss is not due to a single-gene retinal disorder or who have advanced, irreversible retinal loss are unlikely to benefit.
Why it matters
Potential benefit: May allow broader, less invasive gene therapy that preserves vision for people with single-gene retinal disorders and reduces surgical risks.
How similar studies have performed: An FDA-approved subretinal AAV therapy for RPE65 shows gene therapy can work for IRDs, but intravitreal delivery is newer and less proven.
Where this research is happening
Davis, United States
- University of California at Davis — Davis, United States (Active)
Researchers
- Principal investigator: Moshiri, Ala — University of California at Davis
- Study coordinator: Moshiri, Ala
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.