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Gene therapies bring new hope for treatment

Dr Tom Edwards

Dr Tom Edwards

Inherited retinal diseases (IRDs) often strike people in the prime of their life, leading to irreversible vision loss or blindness.

The diseases are caused by genetic ‘mistakes’ which cause the cells of the retina to malfunction – and for many decades patients have been told there is no treatment.

But now the accelerating international pace of gene therapy research is bringing new hope that treatments to stall vision loss, or even partially reverse damage, may be on the horizon.

In Melbourne, CERA Principal Investigator Dr Tom Edwards’ team is investigating a gene therapy for a particular inherited retinal disorder that is common in our region.

“We’re looking at one particular gene and developing a strategy to introduce a correct copy back into the affected retinal cells,” he says.

“We’ve had early success in delivering our gene of interest into cells grown in the lab, which has given us plenty of encouragement.’’

Dr Edwards hopes that, ultimately, he will be able to bring his research to a clinical trial in Melbourne.

“It is an exciting time, we have seen the first approved gene therapy in the US for a variety of retinitis pigmentosa and over the next few years we hope to conduct trials for IRDs here in Melbourne.’’

Dr Edwards says that Melbourne, with its unique combination of vision research and clinical expertise at CERA, the University of Melbourne and Royal Victorian Eye and Ear Hospital, is well positioned to become a Centre for Excellence in Ocular Gene and Cell Therapy.

“Our combined expertise at CERA, the University of Melbourne and the Royal Victorian Eye and Ear Hospital, can give Australians access to world-class research into inherited retinal diseases, and as research progresses, we may develop techniques that can be applied to more common eye diseases with a genetic basis such as glaucoma and age-related macular degeneration.’’


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