iPS Cells of Patients for Models of Retinal Dystrophies

Not Applicable

Recruiting

• Conditions: Retinal Dystrophies
• Interventions: Other: Skin biopsy

• Registration Number: NCT03853252
• Lead Sponsor: University Hospital, Montpellier

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2014-11-03
• Study First Submitted: 2019-02-22
• Study First Submitted QC: 2019-02-22
• Study First Posted: 2019-02-25
• Status Verified: 2022-12
• Last Update Submitted: 2022-12-18
• Last Update Posted: 2022-12-20
• Primary Completion: 2024-11
• Study Completion: 2024-11

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 150

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Skin biopsy	Skin biopsy	-

Primary Outcome Measures

Name	Time	Method
number of human cell models obtained	10 years

Trial Locations

Locations (1)

CNMR Maolya, Genetic Sensory Diseases; 🇫🇷 Montpellier, Occitanie, France