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Gene therapy restores vision in blind patients
Three US patients with Leber's Congenital Amaurosis (LCA) are reportedly enjoying ‘dramatic’ improvements to their sight thanks to gene therapy.
The three have a rare inherited disease caused by mutations in more than ten different genes, which prevent normal function of the retina, the light-sensitive layer of cells at the back of the eye. In the first stage of the clinical trial doctors injected one eye each of 12 patients with an engineered virus carrying the gene RPE65. MRI scans showed the brain could ‘see’ the newly-treated eye. As sight improved, three of the patients received treatment in their other eye. The researchers now hope to treat the second eye of the remaining nine patients, and extend the clinical trial. Principal investigator Dr Jean Bennett, said: "I think it will be a stepping stone to treating more common forms of blindness in both eyes".
Though these are very early stage results in just a few patients, they are highly encouraging, especially following on from similar promise shown by gene therapy for another rare genetic eye disorder, Choroideraemia (see previous news).