Improving gene therapy delivery to the retina.
Official title: Breaching the barrier with matrix biology.
Funded at Upstate Medical University · NIH-10770552 · Data updated 2026-11-12
This study is looking at ways to make it easier for new treatments, like gene therapy and stem cell therapy, to reach the retina by changing the structure of a barrier in the eye, which could help people with retinal diseases get better care.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Upstate Medical University NIH-funded |
| Lab location | 1 site (Syracuse, United States) |
| Project start | February 1, 2023 |
| Project end | January 31, 2026 |
| Funding year | 2024 |
| Award (this year) | $203,750 |
| NIH project number | 5R21EY034696-02 |
| Project ID | NIH-10770552 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates would be individuals suffering from retinal diseases that could benefit from gene therapy or stem cell treatments.
✕Less likely to help
Patients with retinal conditions that do not require gene therapy or stem cell interventions may not benefit from this research.
What this research studies
This research focuses on overcoming the barriers presented by the inner limiting membrane (ILM) of the retina, which hinders effective gene therapy and stem cell treatments. The team aims to manipulate the molecular structure of the ILM to enhance the integration of neural stem cells and viral vectors for gene transfer. They will explore two approaches: genetically altering a key component of the ILM and using a small biomolecule to disrupt its structure. By making the ILM more permeable, the research hopes to improve treatment options for retinal diseases.
Why it matters
Potential benefit: If successful, this research could lead to more effective gene therapies for patients with retinal conditions.
How similar studies have gone: Previous research has shown promise in manipulating barriers for gene delivery, but this specific approach is novel.
Where this research is happening
-
Upstate Medical University
Syracuse, United States Active
Researchers
- Principal investigator: Brunken, William J — Upstate Medical University
- Study coordinator: Brunken, William J
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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.