Gene therapy for blind -born children – can now p

Four blind -born children have had vision improved through gene therapy in London.

Now they can distinguish objects and some have started drawing and writing.

“The results for these children are extremely impressive,” says Michel Michaelides, eye surgeon at the UCL Institute of Ophalmology to BBC.

An experimental gene therapy treatment at Moorfield’s Eye Hospital in London has given four children “life -changing vision improvements”.

The children were born with an aggressive form of the genetic disease Leber’s congenital Amauros (LCA), which causes a rapid deterioration of vision from birth. Previously, they could only distinguish light and darkness, but after the treatment, all parents reported improvements and some of the children can now start drawing and writing.

The new treatment is to inject healthy copies of a defective gene in the back of the child’s eye, very early in life, to treat the disease. One of those who received the treatment is Jace from Connecticut in the United States.

As an infant, his parents noticed that something was not right with Jace’s sight. After several doctor visits and many tests, the family was told that he had a disease caused by a mutation in a gene called Aipl1, something there is no established treatment for.

Heard about the study by chance

But by chance, the family heard about the experimental study in London. The operation went fast and he got four small scars in the eye where healthy copies of the gene were injected into the retina through peep cavity surgery. The healthy genes then start a process that helps the cells in the eye work better and survive longer.

One month after the treatment, the parents noticed that Jace was pushing when strong sunlight poured in through the house windows.

“Before the operation, we could hold up an object in front of him that he did not see. Now he picks up things from the floor and produces toys that he couldn’t do before, ”Jace’s father tells the BBC.

According to the doctors at Moorfields, the test results provide convincing evidence that all four children, from the United States, Turkey and Tunisia, have benefited from the treatment. The research group plans to continue to follow the children to see how long the effects of the treatment lasts.