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‘Life-changing’ therapy treats rare childhood blindness

Four children have gained “life-changing improvements” in sight following pioneering treatment at Moorfields Eye Hospital in London. 

The children were born with a severe impairment to their sight due to a rare genetic deficiency, which means they can barely distinguish between night and day. The gene defect causes retinal cells to malfunction and die. 

However, a new treatment was shown to make retinal cells work better and survive longer. The procedure, developed by scientists at University College London, involves injecting healthy copies of the gene into the retina.

In a trial, four children received the novel therapy in one eye only. All four saw “remarkable improvements” in the treated eye over the following three years, but lost sight in their untreated eye. 

One of the children involved was Jace (pictured), from Connecticut, US. “After the operation, Jace was immediately spinning, dancing and making the nurses laugh,” said DJ, his mum. “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.”

Surgeons are now exploring how to make the treatment more widely available.

Image: Moorfields Eye Hospital (NHS)


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