Toddlers born with the most serious form of blindness in children can now see after a pioneering treatment with gene therapy in an NHS hospital.
The children who suffer from an extremely rare genetic disorder can see shapes, find toys, recognize faces and in some cases can even read and write.
They were born with a serious disorder in their sight because of a rare genetic deficit that influences the AIPL1 gene.
Known as Leber -Congenital Amaurose, the condition is a form of retinal dystrophy, which means that the vision of babies deteriorated very quickly from birth and only has sufficient visibility to distinguish between light and darkness.
Those who are born with the condition are considered legally blind and have until the age of four to be eligible for the 'life -changing' procedure.
It is the first effective treatment in the world for the most serious form of blindness in children and only takes an hour.
The simple procedure includes injecting healthy copies of the affected gene in the back of one eye to 'start' sensitivity.
The 11 were chosen by specialists from Moorfields and UCL Institute of Ophthalmology in 2020 and the operations were performed in Great Ormond Street Hospital.
Toddlers born with the most serious form of blindness in children can now see after a pioneering treatment with gene therapy in an NHS hospital. Jace (right), six, had the treatment when he was two. His father depicted (left)
Jace's operation lasted only an hour with only four small scars in his eye
The family traveled from Connecticut in the United States for the groundbreaking operation
The condition is so rare that children from all over the world had to be found for treatment, in which families from the US, Turkey and Tunisia travel to London.
Jace, from Connecticut in the United States, had gene therapy in London when he was only two years old.
As a young baby, his parents noticed that something was wrong was over his eyesight.
“About eight weeks old, when babies should look at you and smile, Jace didn't do that yet,” his mother DJ told the BBC.
After several visits to doctors and many tests, the family was told that Jace had the ultra rare condition.
“You never think it will happen to you, of course, but there was a lot of comfort and lighting to finally find out … because it gave us a way to move forward,” said Jace's father Brendan.
The family was at a conference on the eye condition when they heard about the experimental test performed in London.
Jace's operation was fast and 'fairly simple', his mother said, with only four small scars in his eyes.
Children depicted a month after their right eye treatment that controls Legoland cars
A child born blind can ride a bicycle after the procedure
The condition is so rare that children from all over the world had to be found for treatment, in which families from the US, Turkey and Tunisia travel to London
The simple procedure includes injecting healthy copies of the affected gene in the back of one eye to 'start' sensitivity
In the first month after the treatment, Brendan Jace Squint noted for the first time when seeing bright sunshine streaming through the windows of their house.
The progress of his son has been 'pretty amazing'.
'Pre-operation, we could have stopped an object near his face and he could not follow it at all.
“Now he picks things off the floor, he takes toys, does things that are driven by his face that he would not have done before.”
“It is really difficult to distinguish the impact of having a little bit of vision,” said Brendan.
Gene therapy was only administered at one eye for four patients to overcome possible safety problems.
Then another group of seven children was treated in both eyes. All 11 had meaningful reactions to the treatment that brought them back to them.
The new genetic medicine was from biotech company Meiragtx.
A child whose left eye was treated can see four years after the procedure
“We are incredibly enthusiastic to see the transforming effect of treatment with each of the young children who have received this genetic medicine,” said Dr. Alexandria Forbes, President and Chief Executive Officer of Meiragtx.
'The proven improvements are unparalleled in treatment benefit compared to any ocular gene therapy at every IRD. These improvements expanded outside the meaningful effects on vision and result in life -changing benefits in all development areas, including communication, behavior, training, mood, psychological benefits and social integration. '
Prof James Bainbridge, Consultant Retinal surgeon at Moorfields and Professor Retinal Studies at the UCL Institute of Ophthalmology said: 'The parents describe the children who get trust in terms of their mobility, their independence, their ability to find their way, also in terms of their recognition of shapes and faces and images.
“Some children can even read and write after the intervention, something that people would definitely not expect in this state, untreated.”
Researchers said the new findings offer hope that children affected by both rare and more common forms of genetic blindness can also benefit from genetic medicine in time.
Further work is done to confirm the early study, which appears in the Lancet Medical Journal.
