Intravitreal gene therapy for inherited retinal disease
Funded at University of California at Davis · NIH-11380542 · Data updated 2026-11-12
A specialized gene therapy approach delivered inside the eye is being studied to help children with inherited retinal diseases maintain or improve vision.
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 | 2026 |
| Award (this year) | $670,650 |
| NIH project number | 5R01EY034123-04 |
| Project ID | NIH-11380542 on NIH RePORTER |
Who could benefit from this research
✓Could help
Pediatric patients (0-11 years old) with inherited retinal diseases who may be eligible for future gene therapy trials.
✕Less likely to help
Patients without inherited retinal diseases or those unlikely to benefit from intravitreal AAV-based approaches may not receive direct benefits.
What this research studies
This grant explores gene therapy strategies for inherited retinal diseases, focusing on ways to deliver therapeutic genes to retinal cells with reduced surgical risk. Researchers aim to overcome limitations of current subretinal injections by exploring intravitreal delivery routes and other AAV-based approaches. The work involves preclinical testing and planning for translating successful strategies to pediatric patients who bear the disease burden. The study could pave the way for safer, broader retinal gene therapies that protect vision in children.
Why it matters
Potential benefit: If successful, this work could lead to safer and more effective gene therapies that preserve or improve sight for children with inherited retinal diseases.
How similar studies have gone: Previous retinal gene therapies have shown promise, with approved treatments for specific mutations, but many approaches remain under investigation and are disease- and delivery-route specific.
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.
-
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.
-
Eye-injection 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.
-
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.