For the first time in the world, childhood blindness was cured with the help of gene therapy in London
Kyiv • UNN
British doctors have successfully performed gene therapy on four children with Leber's congenital amaurosis. After a 60-minute operation, the children were able to see objects, recognize their parents' faces, and even read.
Doctors in the United Kingdom have become the first in the world to cure blindness in children born with a rare genetic disease. This was reported by The Guardian, according to UNN.
Details
It is reported that the children had Leber congenital amaurosis (LCA), a severe form of retinal dystrophy that leads to vision loss due to a defect in the AIPL1 gene. Patients with this diagnosis are legally considered blind from birth.
But after doctors injected healthy copies of the gene into their eyes in a tumor removal surgery that lasted just 60 minutes, the four children can now see objects, find toys, recognize their parents' faces, and in some cases even read and write.
The results of treatment of these children are extremely impressive and show the power of gene therapy in changing lives. For the first time, we have an effective treatment for the most severe form of childhood blindness and a potential paradigm shift towards treatment at the earliest stages of the disease,
In 2020, specialists from Muirfields and UCL selected four children aged one to two years from the United States, Turkey and Tunisia. The surgeries were performed at Great Ormond Street Hospital in London.
Healthy copies of the AIPL1 gene contained in a harmless virus were injected into the retina, the light-sensitive layer of tissue at the back of the eye.
This gene is vital for the functioning of photoreceptors, the light-sensitive cells in the retina that convert light into electrical signals that are interpreted by the brain as vision.
The therapy was performed on only one eye of each patient to avoid possible safety issues. The children were then followed for five years. The results were published in the Lancet.
Professor James Bainbridge, a consultant in retinal surgery at Moorfields and professor of retinal research at the UCL Institute of Ophthalmology, said that children born with LCA can only distinguish between light and dark, and what little vision they have will be lost within a few years.
Some children can even read and write after surgery, which is absolutely impossible to expect in this condition without treatment,
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