Gene therapy: Reducing congenital blindness

Researchers have succeeded, through gene therapy to dramatically improve vision in children who suffer from Leber congenital amaurosis (LCA), a severe genetic disease that usually leads to blindness.

The preliminary phase 1, was conducted in October 2007 in 12 patients aged 8 to 44 years in several countries (USA, Belgium and Italy). The vision improved in all, according to an article published online by the British specialist journal The Lancet. A study had been conducted last year on three young adults.

In children the results were "spectacular", according to the researchers, all children earned sufficient vision to enable them to walk without assistance. The effects were maintained for two years.

A child from birth being able to distinguish only light and shadows could discern the eye color of his father. He could also play with him for the first time in football.

The results of these tests, conducted by Joan Bennett and a team of researchers from the School of Medicine, University of Pennsylvania, were presented at a conference of ophthalmologists in San Francisco.

"This is very exciting for the whole field of gene therapy, said in a statement Katherine High, co-director of the study, which hoped that it could accelerate the development of gene therapy for retinal diseases, such as age-related macular degeneration(AMD).

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