CORDIS Project
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This project focuses on developing a combination gene therapy for rod-cone dystrophies, inherited retinal diseases leading to blindness. By using optogenetic channels and a cone viability factor, the therapy aims to restore light sensitivity and promote the survival of cone photoreceptors in affected individuals.
Rod-cone dystrophies are inherited retinal diseases whose clinical course begin with the degeneration of rod photoreceptors and evolve with the progressive loss of cones that, in turn, leads to complete blindness.
These diseases affect 1:2500 individuals worldwide with many underlying gene defects.
Although gene replacement therapy has been very successful in treating inherited retinal degenerations in the clinic, with one FDA approved product on the market and over 30 clinical trials, it is cos…
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