Abstract
AbstractIn this presentation, we will discuss the last developments of ocular gene therapy aiming to restore visual functions and protect the retina from different forms of inherited retinal dystrophies (IRD). The issue of disease models will also be addressed, especially for Usher syndrome, for which the vector should target the sensory cells of the cochlea and the retina. In addition to gene therapy for recessive and dominant forms of IRDs and the new perspectives to treat them with CRISPR/Cas9 technology, the development of optogenetic tools will be described. Different strategies to restore function of large mutated genes using minigenes or triple vectors to expand the capacity of AAV vectors will also be exposed. The state‐of‐the‐art of the new developing technologies should help to choose the best available tool to attempt to treat the disease of interest.
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