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24 - 11 - 2016

Gene therapy for AMD and retinitis pigmentosa

Neurodegenerative diseases of the retina (AMD, retinitis pigmentosa) lead to blindness. Among the therapeutic research possibilities, there is the possibility to inject a gene coding for a photosensitive protein, to render the cells of the retina phosensitive, thus reproducing partly the activity of the destroyed cones and rods.

Researchers are currently studying a protein called ReaChR. Its gene has been transferred into retina cells of post mortem macaques and humans. It was also transferred in vivo in the mouse. In these three models, ReaChR seems to be effective. In mice, it allowed the reproduction of light avoidance behaviours. The next step is test in humans.