UNRAVELING THE MECHANISM OF RETINAL DEGENERATION AND PHOTORECEPTOR SURVIVAL

Retinal degenerations are the major cause of incurable blindness. The genetic defect has been identified for some of them, however the pathogenic mechanism causing photoreceptor cell death in the retina is still not well known. Understanding the basic processes activating cellular death is fundamental for the development of therapeutic approaches. Our studies are aimed to address some unresolved issues: i) how does the photoreceptor death progress? ii) is degeneration reversible? iii) which molecular mechanism is involved in photoreceptor cell death and will this be a target in therapies? iv) will, in the future, stem cells be a valuable and applicable tool to recover retinal damaged tissue? We will address these questions using new genetic systems in model organisms. To this purpose we generated an innovative murine model for retinitis pigmentosa, a form of retinal degeneration. Detailed studies of this animal model will allow identifying molecules that can be target of pharmacological therapies. Furthermore, we will undertake in vitro studies of retinal stem cells. We believe that understanding retinal stem cell biology is crucial before approaching in vivo transplants in model organisms and before applying these cells as therapeutic tools to recover visual function.