Restoring vision in mouse models of retinal degeneration using human rod opsin.

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Degeneration of the retina is a common cause of blindness. In many cases, this degeneration is predominantly a feature of the rod and cone photoreceptors, leaving the channel of communication between the eye and brain (the optic nerve) intact. In patients with such conditions then, the retina is still present but lacks the ability to respond to light. If one could restore photosensitivity to the retina of these people, one could hope to recreate vision. This indeed has been achieved in some patients using electronic prostheses. This places the emphasis on identifying the best possible way of regenerating visual responses in the degenerate retina. There are a number of exciting technologies in development to achieve this goal. It is likely that each will have strengths and limitations and that different patient groups benefit from different approaches. We are therefore at a stage at which we need to explore a variety of strategies for restoring photosensitivity to the degenerate retina in pre-clinical studies. We recently, trialled a very simple approach to this problem - introducing the protein that makes human rods photoreceptive (rod opsin) to surviving neurones in the retina of a mouse model of blindness. We found that rod opsin could make these neurones photoreceptive, and that the treated mice were able to recognise patterns and natural movies presented with standard computer monitors. If similar results were achieved in human patients with similar conditions, we could hope to allow patients currently completely blind to recognise some objects and use vision for spatial navigation in ordinary indoor lighting. As the intervention involves introducing a native human protein into the part of the body it is ordinarily expressed using methods already in clinical use, safety and ethical barriers to trialling in humans are minimised. However, before moving to patients we need a better appreciation of how this new therapy could work. Here we propose a series of experiments in mice that will tell us how safe and effective this potentially exciting new therapy could be.