Scientific leader
Dr. Deniz Dalkara
In the context of hereditary retinal degenerations, which lead to blindness via the loss of photoreceptors, gene therapy is currently one of the most promising therapies for these unfortunately incurable diseases. In addition to genetic correction approaches (gene addition by gene therapy), so-called mutation-independent strategies, which can be applied to a larger number of patients, are under development. One of these strategies is to preserve vision in these patients by maintaining the normal functioning of cone-type photoreceptors using the expression of a gene encoding a trophic factor. The other complementary strategy using optogenetics is to express a photosensitive protein in the residual cones in order to regain light sensitivity in the advanced stages of the disease. At the Vision Institute, we are working on the translation of these two mutually independent gene therapy strategies to the clinic.
Funding
Vision Institute
Laboratory
Gene therapies and animal models for neurodegenerative diseases
Institut de la Vision
17 rue Moreau
75 012 Paris
