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Success 0f Gene-Therapy in ophthalmology

posted by  in April 30th, 2008  Article Under: Genetics   Tags: , ,   

NEW YORK - Scientists for the very first time have used gene therapy to improve eye sight in people with a mild type of blindness, a development experts called it advance step for the experimental technique.

Some vision was restored in four out of the six young people who were treated with the technique, teams of researchers in the United States and Britain reported Sunday.Two of the volunteers who could only see hand movements were now able to read some lines of an eye chart after two weeks.

Stephen Rose, chief research officer of the Foundation Fighting Blindness said, “It’s a phenomenal breakthrough,” Experts said that technique has the capability to restore blindness from inherited eye diseases.

“I think this is incredibly exciting,” said Dr. Jean Bennett, a professor of ophthalmology at the University of Pennsylvania and a leader of the Philadelphia study.“It’s the beginning of a whole new era of studies.”

Couple of teams of scientists, working apart from each other, each tested gene replacement therapy in three patients, suffering from mild hereditary eye disease called “Leber’s congenital amaurosis,” Until this disease is incurable, which symptoms appears early in infants and causes vision loss and at night blindness.

gene therapy

In gene-therapy the affected gene which causes the vision loss in infants removed from their DNA structure and healthy gene is implanted.And with the passage of time the vision reverse from blindness.The results of the eye experiments have given the field of genetic engineering a boost.

Dr. Ronald Crystal said:

“I think it’s really a big shot in the arm for gene therapy and for medicine in general,” Head of genetic medicine at Weill Cornell Medical College in New York.

Each of the subjects (volunteer) had mutations in a gene, which produces a protein needed by the retina, which senses light and sends images to the brain.Those who lack the gene gradually lose eye sight until they adopt blindness in early adulthood.

On experimental basis, the scientists injected millions of working gene beneath the retina in the back of the eye.Only worst eye was treated, in case of any hazard; the untreated eye was used to compare with the treated eye.After the treatment, their eyesight and light sensitivity were periodically measured.The researchers said, “All three of those treated in Philadelphia showed significant improvement in their vision.”

Among the three volunteers, two were women, 19 and 26, and a man, 26 belongs to Italy. The longest follow-up was six months.Besides reading lines on an eye chart, they could see well even in dim light. Bennett said, “We were not expecting to restore their vision to 20/20.”

No serious side effects reported in patients. In Philadelphia one of the patients found with the hole in retina which didn’t affect his eyesight.The researchers think the hole was due to the surgery and not the injected gene.The researchers said,” there was no evidence has found that the altered virus used to ferry the gene into the retina’s cells had traveled outside the eye to other areas of the body.

The National Eye Institute is funding a third similar study at the University of Florida.

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1 user Feedbacks In " Success 0f Gene-Therapy in ophthalmology "

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Adamant said,

I think we have to concede that although sometimes controversial, gene therapy has a lot to offer. It’s interesting to hear about new developments.

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