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Gene Therapy and Goggles Help Blind Children See
Confirmed
In Short: An international team has used gene therapy and special goggles to help blind children regain some vision.
An international research team has reported new results from the first clinical cohort of blind people treated with an optogenetic therapy for advanced retinitis pigmentosa, according to a study published in Science Advances.
The team followed 15 children, aged from 15 months to 12 years, who were treated with the gene therapy at Great Ormond Street Hospital (GOSH) between 2020 and 2023.
Optogenetic therapy, based on Nobel prize-winning science, involves turning nerve cells on and off using light. The therapy involves inserting a healthy copy of the gene directly into each eye.
Saffie, a child diagnosed with Leber congenital amaurosis (LCA), was one of the patients who received the treatment. Her mother Lisa said the family had been told Saffie would have been 'blind by the age of 30' without treatment.
After undergoing Luxturna therapy at GOSH, Saffie's condition improved significantly. Lisa said, 'Having the gene treatment has been life-changing, it's like someone waved a magic wand and restored her sight in the dark.'
The therapy has allowed Saffie to regain some vision, enabling her to participate in activities she previously could not. Lisa added, 'We couldn't go trick or treating. Life was really, really hard. She missed out on a lot.'
The researchers noted that the therapy is not a cure but can improve sight and strengthen visual pathways at a critical stage of brain development. 'This research highlights not only the potential of gene therapy to change what's possible for children with inherited retinal disease, but also the importance of developing age-appropriate outcome measures,' said a scientist involved in the study.
The optogenetic therapy, which involves wearing special goggles that emit light to activate the treated cells, has shown promising results in clinical trials. 'We’ve gone from one patient to 10 patients, we’ve seen improvement in their light sensitivity … and we’ve seen that we can give a stable restoration of some visual function, some light sensitivity, that lasts for four or five years in these patients,' said a researcher.
The therapy has also shown success in animal models. UC Berkeley researchers injected a gene for a green-light receptor into the eyes of blind mice, and a month later, the mice were navigating around obstacles as easily as mice with no vision problems.
The findings build on a 2021 report that the same approach helped one blind man see and count objects, demonstrating the potential of optogenetic therapy to restore sight in blind individuals.
The researchers are optimistic about the future of this therapy, with plans to continue developing and refining the treatment to help more patients regain their sight.
What this adds
The study adds to the growing body of research on optogenetic therapy, which has shown promise in restoring sight in blind individuals. The therapy involves inserting a healthy copy of the gene directly into each eye, and wearing special goggles that emit light to activate the treated cells.
The therapy is not a cure but can improve sight and strengthen visual pathways at a critical stage of brain development. The researchers noted that the therapy has shown promising results in clinical trials, with patients experiencing improvement in light sensitivity and stable restoration of visual function for up to four or five years.
The therapy has also shown success in animal models, with blind mice navigating obstacles as easily as mice with no vision problems after receiving the treatment.
The researchers are optimistic about the future of this therapy, with plans to continue developing and refining the treatment to help more patients regain their sight.
Background
The team followed 15 children, aged from 15 months to 12 years, who were treated with the gene therapy at Great Ormond Street Hospital (GOSH) between 2020 and 2023.
An international team has used gene therapy and special goggles to help blind children regain some vision.
What's confirmed
- Basel, 7 October 2026 - An international research team has reported new results from the first clinical cohort of blind people treated with an optogenetic therapy for advanced retinitis pigmentosa.
- University of California, Berkeley, scientists inserted a gene for a green-light receptor into the eyes of blind mice and, a month later, they were navigating around obstacles as easily as mice with no vision problems.
- UC Berkeley researchers are hoping to produce a similar therapy for people who are blind, providing enough vision to easily move about and perhaps enough to read or view movies. (Photo by Ehud Isacoff and John Flannery) “You would inject this virus into a person’s eye and, a couple months later, they’d be seeing something,” said Ehud Isacoff, a UC Berkeley professor of molecular and cell biology and director of the Helen Wills Neuroscience Institute.
- Blind mice (top) cautiously keep to the corners and sides, while treated mice (middle) explore the cage almost as much as normal sighted mice (bottom). (Photo by Ehud Isacoff and John Flannery) “To the limits that we can test the mice, you can’t tell the optogenetically-treated mice’s behavior from the normal mice without special equipment,” Flannery said.
- And according to a study out this week in the journal Science Advances, his genes may yield important clues about why we age and possibly how to slow it down.
What's still developing
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- From the age of two, Saffie has worn glasses after being told she was short-sighted.
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