Gene Treatment for Inherited Blindness in Children with LCA5
Gene Therapy for LCA5-Assocated Inherited Retinal Degeneration: Extension to Pediatric Cohorts
['FUNDING_R01'] · UNIVERSITY OF PENNSYLVANIA · NIH-11193462
This research explores a new gene treatment for children and adolescents with a severe inherited eye condition called LCA5, aiming to improve their vision.
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-11193462 on ClinicalTrials.gov |
What this research studies
LCA5 is a severe inherited eye disease that causes significant vision loss from early childhood. This gene therapy aims to correct the underlying genetic defect responsible for the condition. Previous work with this gene therapy in adults with LCA5 has shown promising results, demonstrating both safety and some improvement in vision. This new effort extends the gene therapy to older children and adolescents, specifically targeting those with LCA5-associated inherited retinal degeneration. The goal is to treat vision loss earlier in life, potentially before the brain's visual pathways are fully set, to maximize the potential for vision improvement.
Who could benefit from this research
Good fit: Ideal candidates would be children and adolescents diagnosed with LCA5-associated inherited retinal degeneration.
Not a fit: Patients with other forms of inherited retinal degeneration not caused by the LCA5 gene would not benefit from this specific treatment.
Why it matters
Potential benefit: If successful, this gene therapy could significantly improve vision and quality of life for children and adolescents with LCA5.
How similar studies have performed: Initial results from a similar gene therapy in adults with LCA5 have shown promising signs of safety and effectiveness.
Where this research is happening
PHILADELPHIA, UNITED STATES
- 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 below.