Scientific leader
Dr. Deniz Dalkara
The main objective of this project is to develop new models of nonhuman primates of photoreceptor degeneration, compatible with the evaluation of the safety and efficacy of gene therapies, aimed at visual restoration and transplantation of photoreceptors derived from stem cells expressing a photosensitive microbial opsin. Hereditary retinal degenerations such as pigmentary retinopathy are a major cause of human blindness caused by a large number of mutations in more than 200 genes expressed in the retina – some of which have not yet been identified. These diseases do not appear spontaneously in nonhuman primates, which makes it difficult to evaluate gene and cell therapy approaches in a relevant large animal model with an anatomy and ocular function very close to humans (presence of a macula). Our goal is to create two models with large areas of external retinal degeneration (loss of photoreceptors) compatible with behavioral tests following therapeutic interventions in gene and cell therapy.
Laboratory
Gene therapies and animal models for neurodegenerative diseases
Institut de la Vision
17 rue Moreau
75 012 Paris
