NCT03853252

iPS Cells of Patients for Models of Retinal Dystrophies

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 150 (estimated)
• Sponsor: University Hospital, Montpellier · Academic / Other
• Sex: All
• Age: 5 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

The investigators are focused on inherited retinal dystrophies with an aim to further understand disease pathophysiology and to elaborate novel treatments, as, to date, there is no effective treatment to prevent blindness. The main goal of this study is to generate human cellular models of healthy and disease retinas and perform studies to evaluate the efficiency of gene therapy approaches for different diseases. Skin biopsies of volunteers are cultured to isolate fibroblasts that are then reprogrammed into iPS cells. Healthy and disease-specific iPS cells are then differentiated into retinal models. This study should help to elucidate disease pathways and to provide proof-of-concept for various therapeutic approaches.

Conditions

• Retinal Dystrophies

Interventions

Type	Name	Description
OTHER	Skin biopsy	Skin biopsy on a location preliminarily anesthetized Disinfection protocol Combined required blood tests (HIV, Hepatitis B)

Timeline

• Start date: 2014-11-03
• Primary completion: 2023-10-30
• Completion: 2023-10-30
• First posted: 2019-02-25
• Last updated: 2025-07-10

Locations

1 site across 1 country: France