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World’s First Attempt To Restore Sight Using Gene Therap

uding tests on dogs with LCA, whose vision was restored to the extent they could navigate a maze with ease.

Said Robin Ali, professor of human molecular genetics at UCL: "Testing it for the first time in patients is very important and exciting and represents a huge step towards establishing gene therapy for the treatment of many different eye conditions."

The clinical trial was given a million pounds ($2 million) of funding by Britain's Department of Health, which said the pioneering research underlined the country's leading position in gene therapy in Europe. The idea of using gene therapy to fix diseases caused by genetic faults has long appealed to scientists, although getting the idea to work in practice has proved the tricky part.

Some gene therapy approaches have helped patients. But one 18-year-old volunteer died in a gene therapy experiment in 1999 and two French boys cured of rare immune disease later developed leukemia. Over 70 percent of gene therapy trials to date have been for cancer, where the process is complicated by the need to reach multiple sites in the body.

In contrast, the eye is relatively straightforward according to Andrew George of London's Imperial College. "The eye is good for gene therapy because it is a simple organ and it is easy to see what is going on. There is hope that once gene therapy is developed in the eye, scientists could move on to more complex organs," he was quoted.

Some indications of the result may be available within several months. However, the subjects - Mr. Johnson and the 11 others - will need to be followed up to assess the long term effect of the treatment. It is anticipated that the best results will be achieved in younger patients, as they will be treated when the disease is in its earlier stages of development.
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