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A blind woman in Texas is the first person to undergo therapy based on an emerging technology called optogenetics. If successful, the therapy will create light-sensing cells in one of her eyes and enable her to see again.
This patient and others being recruited for a clinical trial have a degenerative disease called retinitis pigmentosa. In this disease, the light-sensitive cells of the retina gradually die off. These cells pass electrical signals on to nerves that convey them to the brain.
The therapy uses optogenetics, a technology that uses a combination of gene therapy and light to precisely control nerves. The therapy should make certain nerve cells in the woman’s eye, called ganglion cells, light-sensitive. The eye was injected with viruses carrying DNA from light-sensitive algae. If it works, the cells will do what the healthy retina’s cones and rods do: fire off an electrical signal in response to light, restoring some vision.
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Beyond the implications for treating blind people, this trial is also being watched by the neuroscience community. If it’s successful, it suggests that optogenetics has promise not just as a lab tool for studying the brain circuits that underlie diseases like Parkinson’s and schizophrenia, but also as a potential therapy for treating people afflicted with them.
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