April 8, 2025 at 12:55 pm

Groundbreaking New Gene Therapy Restores Sight Of Legally-Blind Children In UK-Based Study

by Kyra Piperides

A child undergoing a sight test at the optician's

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In a groundbreaking new project, a team of UK-based opthamologists have taken huge strides in improving the sight of children born with severe visual impairments.

According to a statement from University College London, the pioneering procedure, which involved keyhole surgery on the eye of the children, saw unprecedented success, with all of the four children involved seeing significant improvements in their sight.

And the results were life-changing, as the mother of one of the children who received surgery at the UK’s world-leading Great Ormond Street Children’s Hospital, confirmed in the statement:

“After the operation, Jace was immediately spinning, dancing and making the nurses laugh. He started to respond to the TV and phone within a few weeks of surgery and, within six months, could recognise and name his favourite cars from several metres away.”

For a child who was born legally-blind, this sudden improvement is astounding.

A girl wearing glasses reading a book

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Each of the children undergoing the new treatment were born with a genetic deficiency that affects the genetic makeup of their eyes, meaning that their sight is severely impaired.

As a result, the retinal cells are unable to properly function and subsequently die.

To combat this, after making a small incision into the eye, doctors injected healthy copies of the affected gene into the retina, allowing the children’s bodies to replace the defective gene and restore the eyesight.

Of course, this surgery has only so far been tested in four children and, due to the risks involved, was only tested on one of each child’s eyes.

Over the course of the next four years, each child’s surgically-treated eye showed incredible improvements in their sight, but their untreated eye lost its sight entirely.

A child choosing glasses in an optician's office

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The study, which was recently published in medical journal The Lancet proved that there is hope for future sight in the lives of children born with these rare visual impairments.

Professor Michel Michaelides – professor of ophthalmology at the UCL Institute of Ophthalmology and consultant retinal specialist at Moorfields Eye Hospital – explained in the statement just how groundbreaking the team’s findings are:

“We have, for the first time, an effective treatment for the most severe form of childhood blindness, and a potential paradigm shift to treatment at the earliest stages of the disease. The outcomes for these children are hugely impressive and show the power of gene therapy to change lives.”

And, given the unprecedented success of their study, ophthalmologists across the world are excited about the prospects for their youngest patients.

With their success in restoring sight to these young children, it is hoped that this gene therapy will be rolled out more widely very soon.

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