Supported projects

V201901 – CRX gene therapy in retinal dystrophies mouse models : proof of concept for CRX dominant mutations as well as CRX independent retinopathies

In recent years, gene therapy in the field of ophthalmology has shown extremely promising results. The objective of our study is to develop a new gene therapy product based on the use of an AAV-virus targeting retinopathies associated with dominant mutations in the CRX gene. Preclinical analysis will be performed in two models of mice with Crx mutations: a Leber congenital amaurosis model, and a cone dystrophy model. Because of the central role of CRX in the regulation of photoreceptor gene expression, we will also test our approach in another mouse model of retinal dystrophy, linked to a CRX target gene.This project will therefore provide necessary data for the development of a clinical trial in retinopathies involving, directly or indirectly, the CRX gene.