Gene therapy for a specific type of inherited retinal degeneration in children
Official title: Gene Therapy for LCA5-Assocated Inherited Retinal Degeneration: Extension to Pediatric Cohorts
Funded at University of Pennsylvania · NIH-10974359 · Data updated 2026-11-12
This study is testing a new gene therapy for teenagers with Leber congenital amaurosis (LCA5), a condition that causes serious vision loss, to see if it can help improve their eyesight before they reach important stages of eye development.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pennsylvania NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | September 15, 2024 |
| Project end | July 31, 2028 |
| Funding year | 2024 |
| Award (this year) | $826,251 |
| NIH project number | 1R01FD008174-01 |
| Project ID | NIH-10974359 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adolescents diagnosed with LCA5 who experience severe vision loss.
✕Less likely to help
Patients with other types of inherited retinal diseases or those who do not have LCA5 may not benefit from this research.
What this research studies
This research focuses on developing a gene therapy for Leber congenital amaurosis (LCA5), a severe inherited retinal disease that leads to early vision loss. The study aims to extend gene augmentation therapy, which has shown promise in adults, to older pediatric patients, particularly adolescents. By using a viral vector to deliver the therapeutic gene directly to the retina, the researchers hope to improve vision outcomes in these young patients before critical developmental windows close. The trial will assess both the safety and efficacy of this innovative treatment approach.
Why it matters
Potential benefit: If successful, this research could significantly improve vision and quality of life for children suffering from LCA5-associated retinal degeneration.
How similar studies have gone: Previous clinical trials for gene therapy in other forms of Leber congenital amaurosis have shown promising results, indicating a potential for success in this approach.
Where this research is happening
-
University of Pennsylvania
Philadelphia, United States Active
Researchers
- Principal investigator: Aleman, Tomas S — University of Pennsylvania
- Study coordinator: Aleman, Tomas S
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.