Gene therapy brings sight to blind
Monday October 26th, 2009
A gene therapy has successfully been used to restore sight to a blind child, it has been announced.
A single injection of genes restored working sight to a boy aged nine, researchers in Philadelphia, Pennsylvania, said.
The boy was faced total loss of sight and already could barely see when he was treated. Now he can join in with normal lessons and ball games.
The
treatment has been used with five children and seven adults with a condition
called Leber's congenital amaurosis and has been reported on-line by The
Lancet. Patients came from Naples, Italy, and Belgium as well as the USA.
Doctors said the greatest improvements involved the children - all of whom can now negotiate a low-light obstacle course.
They said no patient had attained fully normal eye-sight - but half no longer needed to be classified as legally blind.
Researcher Professor Jean Bennett, of The Children's Hospital of Philadelphia, said: "The clinical benefits have persisted for nearly two years since the first subjects were treated with injections of therapeutic genes into their retinas.
"These remarkable results have laid a foundation for applying gene therapy not only to other forms of childhood-onset retinal disease, but also to more common retinal degenerations."
Fellow researcher Dr Albert Maguire said: "In follow-up studies, we will continue to monitor these patients to determine whether this treatment stops the progression of this retinal degeneration.
"In the future, we hope to investigate whether other retinal disease will be amenable to this gene therapy approach."
More details on our blog posting here.
Tags: Child Health | Eye Health | Genetics | North America
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