Newest trial of genetic therapy in London has given the gift of sight to children suffering from blindness since birth.
As reported BBC, an experimental trial of gene therapy has helped in the treatment of four children, who were born with one of the most severe forms of childhood blindness.
The children underwent the therapy at Moorfields Eye Hospital in London, where they received "life-changing improvements" to their sight.
Before the therapy, they could only tell the difference between dark and light, and were registered legally blind.
After the trial, which introduced normal genes into cells in place of missing or defective ones in order to correct genetic disorders, all parents reported improvement – with some of their young children now able to begin draw and write.
Gene therapy for another form of genetics blindness has been available on the National Health Service (NHS) since 2020.
The new work is the continuation of that therapy, where doctors injects healthy copies of a defective gene into the back of the eye, very early in life, to treat a severe form of the condition.
According to Moorfields' doctors, the results of the tests, alongside the parent’s reports of children improvements, give "compelling evidence" that all four benefited from the treatment and showing unexpected improvement.
Consultant eye surgeon, Prof Michel Michaelides, at the UCL Institute of Ophthalmology, added, "The outcomes for these children are hugely impressive and show the power of gene therapy to change lives."
The team of doctors is expected to monitor children progress to see how long-lasting the results are.
