British doctors say they have made the world's first surgical attempt to treat a human sight disorder using gene therapy.
The patient, Robert Johnson, 23, has an inherited disorder called Leber's congenital amaurosis. The disorder, linked to a mutation in a gene called RPE65, is thought to lead to degeneration in the retina — the layer of cells at the back of the eye that detects light.
[...] The doctors — Prof. Robin Ali of the Institute of Ophthalmology at University College London and his colleagues at Moorfields Eye Hospital in London — said they injected non-defective copies of the RPE65 gene under the retina in one of Johnson's eyes.
A harmless virus was used to deliver the gene into the cells.
[...] However, it will be months before the team knows whether the approach worked for Johnson.
If I remember correctly from earlier gene therapy tests, the real trick is whether the genes don't get spliced into the wrong spot and cause nasty cancer. Maybe they've worked around this, which would be great news as there's many nasty diseases that may be treatable by gene therapy.