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UCL in the News: Gene therapy aimed at restoring sight for 30,000 people
2 May 2007
British scientists are to launch the world's first clinical trials of a controversial gene therapy to cure childhood blindness.
Researchers will test the treatment on volunteers with a rare inherited form of blindness, in which a single defective gene causes the retina to degenerate and eventually stop working as the child grows up. …
Patients taking part in the trial will be injected with a virus modified to carry a correct version of the faulty gene. …
The phase 1 trials are designed to assess the safety and efficacy of the treatment in 12 patients and will be carried out by the UCL Institute of Ophthalmology and nearby Moorfields Eye Hospital. …
"If this trial is successful it paves the way for the application of this technology in the treatment of many forms of inherited retinal degeneration and perhaps some of the more common disorders of the eye too," said Professor Robin Ali [UCL Institute of Ophthalmology], who is leading the team.
The researchers will know whether the treatment has worked in a few months. …
Ian Sample, 'The Guardian'
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