Gene therapy clinical trials are underway to treat one of the leading causes of blindness in the developed world.

The treatment for wet age-related macular degeneration (more advanced than dry macular degeneration) will hopefully be available to patients within three years, says the team at the Lions Eye Institute in Western Australia.

They’re using a modified virus to carry a gene into the cells at the back of the eye. The delivered gene encourages these cells to continuously secrete medication to treat the problem.

“It effectively turns the cells into little ‘bio-factories,’” Professor Elizabeth Rakoczy explains.

It’s also more efficient and less invasive than the current treatment, which requires an injection into the eye every month. Elizabeth’s treatment consists of a single injection, under local anaesthetic.

This form of macular degeneration occurs when the proteins in the back of the eye, which are responsible for growing new blood vessels, are overproduced. This causes abnormal growth of blood vessels that are prone to bleeding, damaging the macula and causing vision loss.

Elizabeth believes this is just the beginning for using gene therapy to treat diseases.

“The eye is a nice model to start with, it’s a small organ so it’s easy to monitor what’s going on,” she says.

“Whereas if you’re treating something like MS, you have to work with and monitor many kilograms of muscle around the body.

“Conceptually, this research could change how we treat other complex diseases, like diabetes and heart disease.”

The treatment is 15 years in the making.

“It’s exciting to have taken the research from ‘bench to bed’ – we started from scratch and are now trialling in patients.”

For more information:

Lions Eye Institute
Professor Elizabeth Rakoczy
+61 8 9381 0726
elizabeth.rakoczy@uwa.edu.au
www.lei.org.au/author/erakoczy

Banner image: Helping the blind eye to repair itself using modified viruses.
Credit: Chris Barry