Gene therapy injected into one eye is transmitted to the other eye

Along Claire Wilson

Paul Parker / Science Photo Library

Gene therapy for rare forms of blindness seems to work well, but genes injected into one eye have been shown to be transmitted to the untreated eye. Since such tests usually compare the visual acuity of a treated eye with that of an untreated eye, this finding affects not only safety, but also how to measure the effectiveness of treatment.

Experimental treatment is for a condition called Leber’s hereditary optic neuropathy, which usually affects young men and leads to progression. Blindness.. This is caused by a mutation in one of the genes in the mitochondria, the energy-producing structure inside the cell. It kills cells in the retina, a patch of tissue behind the eye that converts light into electrical signals.

In gene therapy, a harmless virus containing a gene is injected into the eye and taken up by retinal cells. These begin to make the protein encoded by this gene. Mitochondria Helps protect the rest of the retina.

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Eye disorders are a popular target for gene therapy. Because they can only be delivered to one eye, and even if they cause exacerbations, people have untreated eyes. In the latest 37 trials, nearly two years after treatment, 29 of the participants showed at least some improvement in vision in both eyes. Of these, 25 had clinically relevant improvements in the treated eye.

Therefore, according to the original criteria of comparing the visual acuity of the treated eye with the visual acuity of the untreated eye, the treatment failed. The difference in visual acuity improvement was not statistically significant. In future trials, untreated eyes may also be treated. Gene therapy Patrick Yu-Wai-Man of the University of Cambridge says he is with a group receiving placebo injections.

Improvements in both eyes of 29 participants suggest that the virus can leave the treated eye, at least with this form of gene therapy. To figure out what was happening, Yu-Wai-Man and his colleagues injected the virus into one eye of three rhesus monkeys and examined their visual system three months later. They found the virus in both eyes of all three animals. It is also found in their optic nerve, suggesting that it traveled through the optic nerve of the treated eye, entered the other optic nerve where the two nerves intersected, and moved to the untreated eye.

Several other gene therapies have been investigated for different types of blindness, some of which are provided by injection below. retina, May not allow genetic material to move in the same way.

“I don’t know if this is good news or bad news, but it’s fascinating news,” says Michelle Michaelides of University College London. “It’s an important finding to consider when designing a study.”

Journal reference: Scientific translation medicine, DOI: 10.1126 / scitranslmed.aaz7423

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