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Moorfields hospital carries out world's first gene therapy operation to cure blindness
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Â鶹¹ÙÍøÊ×Ò³Èë¿Ú News has learned that a team at Moorfields Eye Hospital in London has carried out the world's first gene therapy operation in an attempt to cure blindness in children and young adults.
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Â鶹¹ÙÍøÊ×Ò³Èë¿Ú Science Correspondent, Pallab Ghosh, has had exclusive access to the patient and the doctors involved in this pioneering operation.
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The procedure was carried out on Robert Johnson, a man who was born with a sight disorder which becomes worse with age. At the moment he can see outlines during the day – but very little when it's dark. As he gets older his vision will become progressively worse.
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Robert told the Â鶹¹ÙÍøÊ×Ò³Èë¿Ú that he hoped that that might change. He is the first person in the world to take part in an attempt to treat blindness using gene therapy. Doctors at Moorfields Eye Hospital have inserted genes into one of Robert's eyes in the hope that his sight might recover.
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On the day of the operation Robert revealed that he had mixed feelings: "It's very difficult to say how I'm feeling, I keep ranging from extreme nervousness to a bit of excitement."
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Robert wasn't the only one who was nervous. His feelings were shared by the lead researcher, Professor Robin Ali, who has spent 15 years working with colleagues developing the technique.
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As Robert was being prepped for his operation, Professor Ali was anxiously waiting in his office at the Institute of Ophthalmology: "I can't help feeling somewhat apprehensive, there's so much riding on it and we've all been waiting for a very long time."
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Robert's disorder is an inherited disorder, caused by a faulty gene called RPE65. This defect stops the layer of cells at the back of the eye from working.
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The operation involves injecting working copies of the genes into the back of Robert's eye, underneath the retina which contains the cells that normally detect light. But in Robert's case it's these cells that are damaged and stop him from seeing properly.
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It is hoped that the replacement genes will enable the retina to detect light – and eventually improve Robert's sight.
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The technique has worked in the lab and it has worked on animals. Asked if he thought it would work on Robert, the surgeon carrying out the operation, James Bainbridge, said:
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"We intend to find out during this experimental procedure. We don't know for sure how someone like Robert's retina is likely to behave in this situation."
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During the operation, James Bainbridge injected normal copies of the gene into the back of Robert's eye. It required incredible precision on the surgeon's part. One slip and Robert's retina would have been torn. The operation would have failed.
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Asked how it went, assisting surgeon, Robert Maclaren said: "Extremely well," whilst beaming through his surgical mask.
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"We couldn't have asked for a better result. In fact if I sat and drew what the perfect result would have been on a piece of paper – the retina would have looked pretty much as it would look now."
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It will be several months before we know if the operation has worked on Robert. He's the first of 12 young people trying out the new technique. If it is successful, the researchers believe that it could be used to treat a wide range of sight disorders.
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Notes to Editors
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Any use of the above should be credited to Â鶹¹ÙÍøÊ×Ò³Èë¿Ú News.
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The exclusive report can be seen first on the Â鶹¹ÙÍøÊ×Ò³Èë¿Ú One O'Clock News.
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