$2,500
total
36 months
Duration
20
Total Visits
18
Spots Remaining
About This Study
This groundbreaking gene therapy trial aims to restore vision in patients with inherited retinal dystrophy caused by RPE65 gene mutations. A single injection delivers a functional copy of the gene directly to retinal cells.
Study Objectives
To assess the safety and preliminary efficacy of gene therapy in improving visual function.
Eligibility Requirements
Inclusion Criteria
- Adults 18-50 years old
- Confirmed genetic diagnosis of RPE65-related retinal dystrophy
- Best corrected visual acuity worse than 20/60
- Sufficient viable retinal cells
- No previous gene therapy
Exclusion Criteria
- Active ocular infection
- Prior retinal surgery
- Immunocompromised status
- Participation in other gene therapy trials
Frequently Asked Questions
Study Location
Scheie Eye Institute
Philadelphia, PA 19104
Map View
Research Team
Dr. Lisa Thompson
University of Pennsylvania
Gene Therapy Program
Study Timeline
Start DateJanuary 1, 2024
End DateJanuary 1, 2027
Enrollment12 / 30