Findings from a first ever gene-replacement clinical trial for choroideremia were described as being ‘promising’ and ‘surpassing expectations’ by Fight for Sight, the charity which part-funded the research through its Tommy Salisbury Choroideremia Fund.
Researchers reported improvements in visual acuity among six patients who received gene therapy for the condition since 2011.
The trial, published last week in The Lancet medical journal, was led by Professor Robert MacLaren, professor of ophthalmology at the University of Oxford. ‘This clinical trial marks a major step in developing gene therapy treatments for retinal diseases,’ said Professor MacLaren. ‘We didn’t expect the results to show such improvements in visual acuity.’
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