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Congenital Form Of Blindness Can Be Cured By Gene Therapy
Youngsters were able to walk unaided around dimly-lit obstacles, take part in lessons at school without extra help, and one child saw the color of his father's eyes for the very first time.
The revolutionary treatment targets a disease of light-catching retinal cells called Leber's congenital amaurosis, or LCA.
Caused by flaws in any one of around 13 key genes, LCA triggers severe loss of vision and abnormal eye movements in early infancy, usually leading to total blindness in the twenties or thirties.
Doctors led by Jean Bennett of the University of Pennsylvania School of Medicine tested a way of tackling this tragedy by inserting a corrective gene in a disabled cold virus.
Their study was published online by The Lancet on Saturday, coinciding with a presentation at a conference of American ophthalmologists in San Francisco.
The modified virus was injected into the eyeball, and infected the diseased cells, in effect, acting like a Trojan horse to deliver the right DNA to the retina.
Twelve patients aged eight to 44 years were recruited in the small, experimental study, and were given the treatment in the eye that had the worst vision.
None of the patients recovered normal sight, but all of them had an at-least 100-fold increase in so-called pupillary light response, meaning the constriction of the pupil when exposed to light.
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