Eye-injection gene therapy for inherited retinal disease
Official title: Intravitreal gene therapy for inherited retinal disease
Funded at University of California at Davis · NIH-11115686 · Data updated 2026-11-12
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 start | July 1, 2023 |
| Project end | May 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $670,648 |
| NIH project number | 5R01EY034123-03 |
| Project ID | NIH-11115686 on NIH RePORTER |
Who could benefit from this research
✓Could help
People (including children) with genetically confirmed single-gene inherited retinal degenerations who meet clinical and safety criteria for AAV-based therapy.
✕Less likely to help
Those whose vision loss is not due to a single-gene retinal disorder or who have advanced, irreversible retinal loss are unlikely to benefit.
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.
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 gone: 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
-
University of California at Davis
Davis, United States Active
Researchers
- Principal investigator: Moshiri, Ala — University of California at Davis
- Study coordinator: Moshiri, Ala
Related research
Other NIH-funded projects on the same topics.
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Integrative Curation of Clinically Relevant Variants in X-Linked Inherited Retinal Disease Genes
A project to expand expert review of genetic variants linked to X‑linked inherited retinal diseases so patients can access gene therapies.
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Intravitreal gene therapy for inherited retinal disease
A specialized gene therapy approach delivered inside the eye is being studied to help children with inherited retinal diseases maintain or improve vision.
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Retinal Disease: Molecular Basis and Pathophysiology
A gene mutation linked to retinal disease is studied to understand how it changes cellular pathways and leads to vision problems.
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Genes that change how inherited retinal disorders behave
This work looks for other genes that change how CRB1-related inherited retinal diseases affect children and adults who carry CRB1 mutations.
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A new antibody drug to restore the retina’s protective barrier
This project tests a novel antibody designed to boost a natural signaling pathway to strengthen the retina’s protective barrier for people with retinal diseases that cause leaking or inflammation.
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Clear genetic answers for X-linked inherited retinal diseases
Building a trusted catalog of gene changes linked to X-linked inherited retinal diseases so people can get more accurate genetic diagnoses and access gene therapies.
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.