'First in the world': London doctors cure blindness in children born with a genetic condition
WORLD | Trending | SCIENCE & TECH | LCA is a severe form of retinal dystrophy that causes blindness due to a defect in the AIPL1 gene (Aryl-hydrocarbon-interacting protein-like 1). People affected by this condition are termed as blind from birth.
Doctors in Britain have become the first in the world to cure blindness in children born with a rare genetic condition called leber congenital amaurosis (LCA). London doctors are using a pioneeringgene therapy for the treatment of such children.
LCA is a severe form of retinal dystrophy that causes blindness due to a defect in the AIPL1 gene (Aryl-hydrocarbon-interacting protein-like 1). People affected by this condition are termed as blind from birth.
Four such children were cured of such blindness after doctors injected healthy copies of the AIPL1 gene with keyhole surgery which took just an hour. The healthy copies of the gene contained a harmless virus which got injected into the retina. The children can now see shapes, find toys, recognise their parents’ faces, and in some cases, even read and write.
'Hugely impressive'
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Prof Michel Michaelides, a consultant retinal specialist at Moorfields Eye Hospital and professor of ophthalmology at the UCL Institute of Ophthalmology called the results of surgery "hugely impressive". He said that the results show the power of gene therapy which can "change lives".
“We have, for the first time, an effective treatment for the most severe form of childhood blindness, and a potential paradigm shift to treatment at the earliest stages of the disease," he told The Guardian.
The four children treated, all aged between one and two, are from the US, Turkey and Tunisia. They were selected for the procedure by specialists from Moorfields and UCL in 2020. Their surgery was carried out at London’s Great Ormond Street Hospital. They were observed for five years and the results were published in the Lancet journal. The therapy was done in only one eye of each patient to avoid any potential safety threat.
Children born with LCA
Prof James Bainbridge, consultant retinal surgeon at Moorfields and professor of retinal studies at UCL Institute of Ophthalmology said that the children born with LCA can only distinguish between light and dark and whatever little sight they possess, they lose it within a few years of birth.
“Some children are even able to read and write following the intervention which is something that one would absolutely not expect in this condition, untreated," he told the British newspaper.
(With inputs from agencies)
