Gene therapy can be particularly effective in treating inherited sight problems in children, fresh trials show.
US doctors treating 12 patients with a rare genetic eye disorder were able to significantly improve vision in the youngest.
The study, published in The Lancet, builds on work carried out by doctors at London's Moorfields Eye Hospital.
The eye is proving to be a particularly successful target for gene therapy.
The principle is simple: to replace a defective gene and restore function to a part of the body affected by a genetic disorder.
Striking early
Treatment so far has focused on Leber's congenital amaurosis (LCA), a rare inherited disorder which causes gradual deterioration in vision and can lead to blindness by the time the patient is 20.
It occurs when faulty genes, called RPE65, stop the layer of cells at the back of the eye working.
LCA affects approximately one in 80,000 people, and is responsible for one in 10 severe sight disorders in children. Read more...
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