In a medical first, doctors in London have helped four young children born blind to see — and it’s all thanks to a game-changing new gene therapy.
These children were born with a rare eye condition called leber congenital amaurosis (LCA). It’s a genetic disorder that causes severe vision loss from birth. Until now, there was no effective treatment for it.
But thanks to a team of specialists from Moorfields Eye Hospital, UCL Institute of Ophthalmology, and Great Ormond Street Hospital, that’s no longer the case.
A Simple Procedure, Life-Changing Results
Here’s what happened: doctors used a special form of gene therapy to inject healthy copies of a missing gene into the back of the children’s eyes. The gene, called AIPL1, is important for cells in the eye that respond to light — the kind that help us see shapes, colors, and movement.
The treatment was done using keyhole surgery, a minimally invasive method that only took about an hour. Only one eye was treated in each child to make sure the therapy was safe.
And the results? Nothing short of incredible.
The kids, aged between one and two at the time of surgery, can now:
- See and recognize shapes
- Spot toys across the room
- Recognize their parents’ faces
- And in some cases, even read and write
Professor Michel Michaelides, one of the leading doctors on the project, called the outcomes “hugely impressive” and said this could change how we treat childhood blindness in the future.
From No Reaction to Light — To Stealing Phones
Take Jace, for example. He was two years old when he got the treatment in 2020. Before the surgery, he couldn’t track anything with his eyes — not even bright or colorful objects right in front of his face.
But within a month, his parents noticed something different. When sunlight came through a window, Jace flinched and pulled back — his first real reaction to light. That moment brought tears to his dad’s eyes.
Now, Jace is six years old and can pick up tiny things off the floor, recognize toys from across the room, and even swipe phones out of his teachers’ pockets (which his parents find pretty hilarious).
His mom DJ called the transformation “pretty amazing.” The family had traveled all the way from Connecticut, USA, for the treatment — and they say it was absolutely worth it.
What’s Next?
Since this success, another seven children have been treated with the same therapy at Evelina London Children’s Hospital. The results of the first trial were published in The Lancet, a respected medical journal, and more long-term results will be tracked for five years.
The therapy was developed by researchers at UCL and made possible through a special license from the UK’s Medicines and Healthcare Regulatory Agency (MHRA), with support from gene therapy company MeiraGTx.
This is just the beginning — and for many families around the world, a ray of hope.
Source: https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(24)0281…
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