Gene Therapy Restores Sight in People with Eye - TopicsExpress

Gene Therapy Restores Sight in People with Eye Disease. Choroideremia is caused by defects in the CHM gene, which produces a protein called REP-1 and affects one in 50,000 people. In those who have the disease, a lack of REP-1 means that cells in the retina stop working and slowly begin to die off, causing blindness. When he was in his twenties, doctors told Wyatt that he would be blind by the time he was 50 – and that there was no cure. Robert MacLaren of the University of Oxford and his colleagues decided to see if it could correct choroideremia. And today the team reports that, of the six people who received the treatment six months ago or longer, all have described improvements in their vision. The very next day I saw a mobile phone and I said I can read the digits! I hadnt been able to read the digits on a mobile phone for five years, says Wyatt. All the people in this trial had varying levels of degeneration before the treatment. However, MacLaren is hopeful that the therapy could also be used to stop choroideremia before there is any significant loss of vision.

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