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Clinical Trial 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.


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT03853252
Study type Interventional
Source University Hospital, Montpellier
Contact Vasiliki Kalatzis, PhD
Phone (0)499636097
Email vasiliki.kalatzis@inserm.fr
Status Recruiting
Phase N/A
Start date November 3, 2014
Completion date November 2024

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