OnMedica – News – Gene therapy focuses on AMD

An Oxford woman participates in early therapeutic trials to prevent vision loss

An Oxford woman has become the first person in the world to benefit from gene therapy in an attempt to end age-related macular degeneration (AMD).

Surgeons have injected a synthetic gene behind Janet Osborne's eye to prevent the death of more cells. This is the first treatment to target the underlying genetic cause of AMD, which affects approximately 600,000 people in the UK.

Osborne, 80, is the first of 10 patients with dry AMD to participate in a gene therapy therapy trial, made by Gyroscope Therapeutics, funded by Syncona, the venture capital arm of the Wellcome Trust.

An injection at the back of the eye releases a harmless virus containing a synthetic gene. The virus infects retinal cells and releases the gene. This allows the eye to make a protein designed to prevent cells from dying and thus keep the macula healthy.

The early-stage trial, at Oxford Eye Hospital, is aimed primarily at verifying the safety of the procedure. It is underway in patients who have already lost some of their vision. If successful, the goal would be to treat patients before they lose their sight, with the goal of stopping AMD.

The treatment was performed under local anesthesia last month at the Oxford Eye Hospital by Robert MacLaren, professor of ophthalmology at Oxford University.

He told the BBC: "Genetic therapy administered early to preserve the vision of patients who lose their sight would be a great advance in ophthalmology and certainly what I hope to see in the near future."

It is too early to know if Ms. Osborne's vision loss to her left eye has been stopped, but the vision of everyone involved in the trial will be monitored. Osborne told BBC News: "I find it difficult to recognize faces with my left eye because my central vision is blurry, so if this treatment could stop it from getting worse, it would be amazing."

Last year, doctors at Moorfields Eye Hospital, in London, restored the sight of two AMD patients by implanting a stem cell patch on the damaged area at the back of the hospital. # 39; s eye. It is hoped that stem cell therapy could help many people who have already lost their sight.

But the Oxford test is different because it aims to address the underlying genetic cause of AMD and could be effective in curbing the disease before people become ill. blind.