How retinal cells and vision respond to gene therapy
Retinal structure, function and response to gene therapy
['FUNDING_R01'] · UNIVERSITY OF PENNSYLVANIA · NIH-11290411
This project looks at how retinal cells and vision change in people with inherited retinal disorders, including after gene therapy such as Luxturna.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF PENNSYLVANIA (nih funded) |
| Locations | 1 site (PHILADELPHIA, UNITED STATES) |
| Trial ID | NIH-11290411 on ClinicalTrials.gov |
What this research studies
If you join, you will have very high-resolution imaging that can see individual cone and RPE cells in your retina. You will also do brief tests of how those cells are working using microperimetry and optoretinography. The team will follow people with different inherited retinal diseases over time to see how the retina normally changes and how it changes after gene therapy. For people treated with Luxturna, the study will measure structural and functional responses in the treated areas.
Who could benefit from this research
Good fit: People with inherited retinal degenerations—especially those with RPE65-related disease or other cone/rod disorders—who can travel for imaging and follow-up are ideal candidates.
Not a fit: People with non-genetic retinal disease or very advanced retinal cell loss may not get direct benefit from the interventions studied.
Why it matters
Potential benefit: Findings could help doctors pick the best time and place in the retina to give gene therapies and improve chances of vision benefit.
How similar studies have performed: Gene therapy like Luxturna has helped some people with RPE65 disease, and high-resolution imaging and functional measures are promising tools that are still being refined.
Where this research is happening
PHILADELPHIA, UNITED STATES
- UNIVERSITY OF PENNSYLVANIA — PHILADELPHIA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: MORGAN, JESSICA I.W. — UNIVERSITY OF PENNSYLVANIA
- Study coordinator: MORGAN, JESSICA I.W.
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.