Understanding how the body reacts to gene therapy for eye diseases
Official title: Elucidating Host Responses to AAV Ocular Gene Therapy Vectors
Funded at Harvard Medical School · NIH-11003698 · Data updated 2026-11-12
This study is looking at how the immune system reacts to a type of gene therapy used to treat eye diseases like inherited blindness, with the goal of finding ways to make these treatments safer and more effective for patients.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Harvard Medical School NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | February 1, 2024 |
| Project end | January 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $36,918 |
| NIH project number | 5F31EY035867-02 |
| Project ID | NIH-11003698 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with inherited forms of blindness or other ocular diseases that may benefit from AAV gene therapy.
✕Less likely to help
Patients with non-genetic causes of blindness or those who do not have access to AAV-based therapies may not benefit from this research.
What this research studies
This research investigates the body's immune responses to adeno-associated virus (AAV) vectors used in gene therapy for treating eye diseases, particularly focusing on conditions like inherited blindness. The study aims to understand the mechanisms behind inflammation and toxicity that can occur after treatment with AAV-based therapies, such as Luxturna. By examining the signaling pathways activated by these vectors, the researchers hope to identify ways to mitigate adverse effects and improve the safety and efficacy of gene therapies for patients. The research will involve laboratory experiments using animal models to explore these responses in detail.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective gene therapies for patients with inherited eye diseases.
How similar studies have gone: Previous research has shown success with AAV gene therapies, but this specific investigation into immune responses is relatively novel.
Where this research is happening
-
Harvard Medical School
Boston, United States Active
Researchers
- Principal investigator: Gardner, Apolonia — Harvard Medical School
- Study coordinator: Gardner, Apolonia
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.