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Inherited blindness can now be cured with gene therapy

A breakthrough gene therapy restores eyesight in patients with rare eye disease


Key points from article :

Jake Ternent has been gradually losing his central vision since birth.

His condition, leber congenital amaurosis, is caused by two faulty copies of a gene RPE65.

The NHS agreed to fund the treatment, Luxturna, the first new generation gene therapies for conditions causing blindness.

Jake says that not only has it stabilized the sight in his right eye, it also reversed some of the decline in vision.

Injection delivers working copies of RPE65 into the retina.

The DNA is encased in a harmless virus which breaks into retinal cells.

Once inside the nucleus, replacement gene kick-starts production of the protein essential for healthy vision.

Matthew Wood, 48, has the same retinal condition and lost his central vision 10 years ago.

Since receiving the gene therapy last year, he noticed subtle, but welcome, changes in his vision.

Matthew hopes his second gene therapy will prevent any further loss of sight.

Children to benefit most as they can be treated before permanent damage is done.

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UK National Health Service, publicly funded healthcare system in England


Global medicines company for developing and producing drugs.

Topics mentioned on this page:
Gene Therapy, Vision (health)