Gene therapy for a common cause of blindness.
Official title: Preclinical evaluation of a homing endonuclease gene therapy for adRP in models of P23H retinopathy.
Funded at University of Louisville · NIH-11053608 · Data updated 2026-11-12
This study is testing a new gene therapy to help people with a specific type of inherited vision loss called autosomal dominant Retinitis pigmentosa, focusing on a common genetic change, and it aims to see if this treatment can stop vision loss and keep the eyes healthy.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Louisville NIH-funded |
| Lab location | 1 site (Louisville, United States) |
| Project start | March 1, 2023 |
| Project end | February 29, 2028 |
| Funding year | 2025 |
| Award (this year) | $574,292 |
| NIH project number | 5R01EY032645-03 |
| Project ID | NIH-11053608 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with autosomal dominant Retinitis pigmentosa, particularly those with the P23H mutation.
✕Less likely to help
Patients with other forms of retinal degeneration or those without the P23H mutation in the rhodopsin gene may not benefit from this research.
What this research studies
This research investigates a novel gene therapy approach to treat autosomal dominant Retinitis pigmentosa (adRP), specifically targeting the P23H mutation in the rhodopsin gene. Using a meganuclease genome editing tool, the therapy aims to correct the genetic defect in preclinical animal models, particularly in pigs that closely resemble human retinal structure. The treatment involves delivering the gene-editing tool via subretinal injection and assessing its effectiveness in halting retinal degeneration and preserving vision. The study will also evaluate the safety of the approach by ensuring it does not cause unintended genetic changes.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment that halts or reverses vision loss in patients with adRP.
How similar studies have gone: While gene therapy for retinal diseases is an emerging field, this specific approach using meganuclease editing for the P23H mutation is novel and has not been extensively tested in clinical settings.
Where this research is happening
-
University of Louisville
Louisville, United States Active
Researchers
- Principal investigator: Mccall, Maureen a — University of Louisville
- Study coordinator: Mccall, Maureen a
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.