Blind Patient Recovers Partial Vision with Optogenetics

After receiving an intraocular injection of the gene for a light-sensitive protein, a 58-year-old man diagnosed with the neurodegenerative eye disease retinitis pigmentosa was able to locate objects on a table using engineered goggles.

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ince its early days in the mid-2000s, optogenetics, with its potential to activate neurons with light, emerged as a promising technique for restoring vision in blind patients. Patients who were blind or nearly blind from retinitis pigmentosa could detect light and motion following treatment. The 58-year-old man described in the paper was the first patient in a clinical trial—partially funded by GenSight Biologics—designed to assess the safety and, secondarily, the efficacy of the therapy. Forty years before enrolling in the trial, he had been diagnosed with retinitis pigmentosa, a rare, genetic disease that results in the degeneration of the photoreceptor cells in the retina, leading to blindness.

To compensate for the loss of these photosensitive cells, the team sought to make existing retinal ganglion cells—which in healthy conditions receive, via other intermediate cells, information from photoreceptors—respond to light. “We do this by taking light sensors from microbes and [delivering them] to the blind retina using gene therapy,” said Roska in a press briefing last week.

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