Doctors in London cure blindness in children with rare condition
Guardian1 day
UK Doctors Advance Gene Therapy to Address Childhood Blindness
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Image copyright: BSIP/Contributor via Getty Images
The Facts
Doctors at Moorfields Eye Hospital and the UCL Institute of Ophthalmology have administered the world's first gene therapy treatment for children with severe retinal dystrophy caused by the AIPL1 gene defect — performing the procedure on four children aged between one and three years old in 2020.
The treatment involves a one-hour keyhole surgery where healthy copies of the AIPL1 gene, contained within a harmless virus, are injected into the retina at the back of one eye, enabling the light-sensing cells to function properly and survive longer.
Before treatment, the children could only distinguish between light and dark. Following the procedure, they gained the ability to see shapes, find toys, recognize faces, and, in some cases, they developed reading and writing skills, with improvements sustained over a three to four-year follow-up period.
The Spin
Narrative A
This is the first effective intervention for the most severe form of childhood blindness — highlighting the transformative power of early genetic intervention in preserving and improving vision and potentially opening doors for treating other forms of genetic blindness in the future.
Narrative B
While this treatment has enabled children to gain independence, confidence, and mobility they never had before, fundamentally changing their daily lives and developmental potential, gene therapy isn't always permanent. More research is needed to make it long-lasting and more widely available.
