
Experimental gene therapy using optogenetics has helped partially restore visual function in patients with severe hereditary blindness - retinitis pigmentosa.
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Retinitis pigmentosa is a hereditary disease that leads to the destruction of retinal cells and blindness.
Experimental gene therapy has helped partially restore vision in people with severe hereditary blindness. After treatment, some patients were able to better distinguish light and determine the location of objects. The results of the scientists' study were published in the New England Journal of Medicine (NEJM).
The study involved 10 people with retinitis pigmentosa, a hereditary disease that gradually destroys the cells in the retina that process light. All participants almost completely lost their vision.
Scientists introduced a special gene into the patients' eyes, thanks to which the remaining retinal cells became sensitive to light. For this purpose, the optogenetics method was used. After the procedure, participants wore special glasses with a camera, which converted the surrounding image into light signals perceived by the modified cells.
After treatment, seven out of ten patients had improved sensitivity to light, with six changes being clinically significant. Some participants became better at noticing objects, determining their location, and more accurately reaching for them with their hands. In four people, improvements were maintained over months and years of follow-up.
The study of brain activity also showed that visual information entered the visual cortex and was processed by it. This means that even after long-term vision loss, the brain is able to perceive new visual signals.
However, it was not possible to completely restore vision: the patients still could not see or read normally.

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