Leber’s Congenital Amaurosis (LCA) is a type of inherited retinal degeneration. The defect prevents normal function of the retina, the light-sensitive layer of cells at the back of the eye. The RPE65 gene is abnormal in those with the disease. People with LCA usually lose vision from infancy.
For almost 15 years and along with colleagues, researchers at Moorfields Eye Hospital in London have been developing a gene therapy to treat this disorder. Dogs affected by the eye ailment were treated with this technique and had their vision improved so that they could walk through a maze, something they were previously unable to do. Today’s human trials are a first.
“Testing it for the first time in patients is very important and exciting and represents a huge step towards establishing gene therapy for the treatment of many different eye conditions,” Robin Ali, professor of human molecular genetics at University College London, said in a statement.
The therapy used in the trial involves inserting healthy copies of the RPE65 gene into the cells of the retina to help them function normally. Restoring the activity in these cells should restore vision.
Some of the patients in the study have had the treatment. It will be many months yet before the scientists know whether or not the treatment was successful. But, Professor Ali adds, “The results from this first human trial are likely to provide an important basis for many more gene therapy protocols in the future, as well as potentially leading to an effective treatment for a rare but debilitating disease.”
University College London (05/01/07)
ABC News (05/02/07)
The New Zealand Herald (05/03/07)
The Times of India (05/03/07)
Herald Sun (05/03/07)
Medical News Today (05/03/07)
Optician Online (05/04/07)