UK doctors have become the first in the world to successfully cure blindness in young children born with a rare genetic illness called Leber Congenital Amaurosis-4 (LCA4).
After being practically blind from birth, four toddlers treated with ground-breaking gene therapy are now able to recognise people, locate toys, distinguish shapes, and in certain situations, read and write, according to doctors at Moorfields Eye Hospital in London.
To “kick start” a process which helps the cells at the back of the eye work better and survive longer, healthy copies of the faulty gene are injected into the back the eye during surgery, according to the results published in the Lancet Journal.
The operation of photoreceptors, which are light-sensing cells in the retina that translate light into electrical signals that the brain interprets as vision, depends on this gene, according to the journal report.
Prof Michel Michaelides, a consultant retinal specialist at Moorfields Eye hospital and professor of ophthalmology at the UCL Institute of Ophthalmology, said : “The outcomes for these children are hugely impressive and show the power of gene therapy to change lives.”
To determine how long-lasting the effects are, the team will continue to monitor the four children treated.
Experts hope that the next step will be a licensed treatment, and be more freely accessible in the UK and other countries.
The first children to receive the therapy were from the US, Turkey and Tunisia.
The NHS has offered gene therapy for a different type of hereditary blindness since 2020.