Warm adhesive gel to help deliver gene therapy to the retina
Retinal-adhesive thermoresponsive gel for AAV-mediated gene delivery to the outer retina
This uses a sticky, temperature-sensitive gel to help deliver AAV gene therapy to the outer retina for people with inherited or degenerative retinal diseases.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pittsburgh at Pittsburgh NIH-funded |
| Lab location | 1 site (Pittsburgh, United States) |
| Project ID | NIH-11163428 on NIH RePORTER |
What this research studies
Many gene therapies for retinal disease rely on delicate subretinal injections that can damage remaining photoreceptors, especially near the fovea. This project develops a thermoresponsive gel that becomes adhesive at body temperature and can carry AAV viral vectors to the outer retina to reach photoreceptors and the retinal pigment epithelium. Researchers will optimize the gel formulation and test how well it spreads, targets the right cells, and limits inflammation in lab and preclinical models. The aim is to create a safer, more effective delivery method that could broaden gene therapy options for various retinopathies.
Who could benefit from this research
Good fit: Ideal candidates would be people with inherited or degenerative retinal conditions affecting photoreceptors or the retinal pigment epithelium who are potential AAV gene therapy recipients.
Not a fit: People without retinal disease, those whose vision loss is from non-genetic causes, or patients with end-stage retinal degeneration lacking viable photoreceptors are unlikely to benefit.
Why it matters
Potential benefit: If successful, this could make retinal gene therapies safer and better at reaching the central retina, potentially preserving or improving vision for people with certain retinal degenerations.
How similar studies have performed: FDA approval of Luxturna shows retinal gene therapy can work, but using an adhesive thermoresponsive gel for AAV delivery is a novel approach that remains largely at the preclinical stage.
Where this research is happening
Pittsburgh, United States
- University of Pittsburgh at Pittsburgh — Pittsburgh, United States (Active)
Researchers
- Principal investigator: Sahel, Jose-Alain — University of Pittsburgh at Pittsburgh
- Study coordinator: Sahel, Jose-Alain
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.