Doctors in London cure blindness in children with rare condition | Blindness and visual impairment
Doctors in London became the first to treat blindness in children born in a rare genetic condition using a pioneering genetic treatment.
Children had congenital matters (LCA), a sharp form of retinal atrophy that causes visual loss due to a defect in the AIPL1 gene. The affected people are legally accredited to be blind from birth.
But after doctors have achieved healthy copies of the gene in their eyes with only 60 -minute key hole surgery, four children can now see shapes, find games, learn about their parents ’faces, and in some cases, even read and write.
“The results of these children are very impressive and the strength of genetic therapy appears to change lives,” said Professor Michel Michaelds, a retinal consultant at MOORFILDS for Eye Hospital and Ophthalmology Professor at the Eye Medicine Institute of Basic Medicine.
“We have, for the first time, an effective treatment for the most dangerous form of childhood blindness, and the potential model turns into treatment in the early stages of the disease.”
Four children between one and two of the United States, Turkey and Tunisia were chosen by MOORFILDS and UCL specialists in 2020. Operations were carried out at Great Ormond Street Hospital in London.
Healthy copies of the AIPL1 gene, mentioned in an harmless virus, were injected into the retina, the light tissue layer of light in the back of the eye.
The gene is vital for the function of optical receptors, and the rented cells of the light in the retina that converts light into electrical signals explained by the brain as a vision.
Treatment is only given in one eye for each patient to overcome any possible safety problems. Then follow the children for five years. The results were Posted in Lancet magazine.
Professor James Pinberridge, a network surgeon consultant at MOORFILDS and professor of retinal studies at the UCL Institute of Eye Science, said that children born with LCA can distinguish only light and darkness, and what a little scene will lose within a few years.
“Even some children are able to read and write after intervention, which is something that one does not expect at all in this case, without treatment.”
One of the children’s parents described the results as “very amazing” and said they felt “lucky” to benefit. Their six -year -old son, who was at the age of two, could choose small things from the ground and select games at a distance.
Brendan and DJ, who did not want to participate their title, traveled from Connecticut in the United States for treatment in September 2020.
“Before the surgery, about two years old, you could carry any object, even a few inches on the face of Jas, and he will not be able to follow him,” said DJ. “It does not matter how bright it is, what is the color, and its shape.
“Now we get calls and notes from school from school, as it steals phones from the pockets of the background teachers, which is hysterical to us.”
Brendan said he had noticed a difference during the first month when his son’s reaction to the bright sun was through a window. He pulled a kind of himself. It was not even closed eye, it was more than a physical reaction.
“I remember that I am really dealing with passion because that was the first time that JACE had any reaction to any kind of light stimulation or anything like that. From there, it was very great.”
UCL developed the treatment using a special license that the Organizational Agency for Medicines and Healthcare (MHRA) and the support of the MeyergtX genetic therapy company.
Since the four children received treatment, seven others were treated at Evlina London for Children’s Hospital by specialists from St. Thomas Hospital, Great Ormond Street and Mooorfields.
