The impact of network remodelling on outcomes for regenerative medicine in the retina - defining the therapeutic window

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Inherited retinal diseases are a leading cause of blindness, affecting millions of people worldwide. These conditions typically lead to a loss of the specialised light sensing cells of the eye progressing to a loss of vision and ultimately blindness. There are a number of promising regenerative approaches to treating these conditions currently in human clinical trials, raising the possibility of restoring vision to many human patients. Visual loss in these conditions is generally a slow, progressive process, yet the current clinical trials are confined to those patients in which all vision has been lost. The diseases involve complex slow changes to the visual tissues and there is reason to believe that the timing of regenerative interventions may be critical in determining the ultimate success of therapy. Here we have devised a program of experiments that determine the impact of the time of intervention upon the level of visual restoration using an established animal model of the human condition. We will use one of the promising current optogenetic approaches to regenerative therapy delivered to a mouse model of hereditary human blindness. The treatment will be delivered at different stages of the disease and its ability to restore vision will be assessed. In order to understand the process at the cellular level we will measure the detailed responses of the visual system in neurons in the retina and brain using state of the art approaches. These findings will influence the development of new improved treatment options for patients suffering from retinal disease and loss of vision.