Using cell surface antigens for the isolation of photoreceptor precursor cells for retinal stem cell therapy

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Diseases of the retina resulting in the death of the light-sensing photoreceptor cells are the leading cause of blindness in the developed world. These conditions affect 1 in 3000 people from birth and more than 10% of the ageing population. They are currently untreatable and irreversible. We recently demonstrated in proof-of-concept experiments in mice that replacing lost photoreceptors by cell transplantation is a feasible clinical treatment strategy. We discovered that by transplanting immature photoreceptor cells, these cells can make new functional photoreceptors in the diseased retina, and restore some visual function. To translate our findings to a human therapy we need to isolate equivalent human photoreceptor precursors from stem cell cultures grown in the laboratory. Our hypothesis is that retinal cells generated from stem cell cultures will be effective and safe for human therapy provided they are treated so that they reach exactly the right stage in a cell culture dish and provided only these correctly-staged cells are transplanted. One critical challenge therefore is developing methods to identify and collect correctly-staged donor cells to transplant into the diseased retina. We will develop methods for isolating live cells by using antibodies that bind to special markers on the surface of the immature photoreceptor cells. The efficiency of the newly isolated cells for retinal repair will be tested in transplantation experiments. This research will develop cell isolation protocols for human photoreceptor precursor cells, which is an essential step towards development of clinical trials for incurable retinal diseases causing blindness.