AAV-MEDIATED RETINAL GENE TRANSFER IN ANIMAL MODELS OF RETINAL DEGENERATION

Retinal gene transfer is a promising approach to elucidate the basis of inherited retinal degenerations in vivo and to design successful therapeutic strategies for a group of diseases for which no therapy is currently available. Despite the genetic heterogeneity of inherited retinal degenerations including retinitis pigmentosa (RP), common mechanisms, like apoptosis, are responsible for photoreceptor cell death. This allows to test strategies aiming at retarding or arresting apoptosis independently from the mutation causing the disease, potentially representing a one-fits-all solution. In addition, the high recurrence of some RP-causing mutations (i.e. the rhodopsin P23H) allows to design experimental strategies that, although specific for one mutation, are related to the disease affecting a large number of individuals. This project aims at developing adeno-associated virus (AAV)-based gene transfer strategies to inhibit photoreceptor cell death in the retina of animal models of RP.