Gene therapy new frontier in fight against glaucoma

Melbourne researchers are turning to gene therapy in the fight against glaucoma, a disease of the optic nerve which affects 60 million people worldwide.

Centre for Eye Research Australia Managing Director and Ringland Anderson Chair of Ophthalmology at the University of Melbourne Professor Keith Martin is leading research to develop new therapies to save the sight, and even restore lost vision, in people suffering from glaucoma.

Glaucoma is caused by damage to the optic nerve which connects the eye to the brain. It plays an essential role in our vision, allowing the brain to receive electrical signals from the back of the eye, so it can interpret them as images.

Glaucoma progression can often be slowed or stopped by lowering eye pressure but about 15 per cent of patients continue to lose sight despite currently available treatments.

“Gene therapy is offering new potential and hope for patients whose glaucoma does not respond to conventional treatments,” says Professor Martin.

“Gene therapy to treat eye disease is advancing at a faster pace than arguably in any other branch of medicine.”

In its simplest form, gene therapy for eye disease works by identifying a defective gene which causes vision loss, producing a correct copy in the lab and reintroducing it to the use using a specially-engineered virus.

The worlds’ first ocular gene therapy, for a rare inherited retinal disease, has been approved for clinical use in the US and europe – and it is anticipated that many more gene therapies for other rare single-gene diseases will become available in the next few years

“As this technology develops, we will see the development of techniques that can be applied to more common eyes diseases such as glaucoma,” says Professor Martin.

Professor Martin, who arrived in Melbourne from the University of Cambridge this year, was involved in the development of a gene therapy for glaucoma which is now heading towards clinical trials in the US.

His work in Melbourne will see his team focus on developing new therapies to repair the optic nerve, ultimately aiming to improve the transmission of signals to the brain and restore vision.

Restoring vision may be a long way off – but these early days are showing it is a realistic possibility for the future.

“We are on the brink of an exciting new era in vision research where restoring sight – once considered impossible is now a realistic goal in the coming decade,” he says.

Professor Keith Martin will present the David Danks Oration at the University of Melbourne on Wednesday 18 September 2019 from 6- 7pm.

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