Gene therapy mends optic nerves in promising advance for treating glaucoma

By Nick Lavars November 05, 2020

A breakthrough in gene therapy has raised hopes of new treatments for glaucoma, a leading cause of vision loss

Researchers in Australia are claiming an important breakthrough in glaucoma research, demonstrating how the degenerative condition can be treated using an innovative form of gene therapy. The technique was shown to be effective at repairing and protecting damaged optic nerves with unprecedented effectiveness, raising hopes of tackling the irreversible form of vision loss that affects millions worldwide.

As the leading cause of irreversible blindness in the US, glaucoma takes hold as the nerve linking the brain and eye endures ongoing damage, usually due to high pressure in the eye, causing a breakdown in the relay of visual information. This type of injury to optic nerve cells cannot be repaired, though further damage can be avoided through various treatments.

At the center of this new breakthrough is a protein called protrudin, and the gene responsible for producing it. Protrudin is normally found in low levels in non-regenerative neurons, and the research team, from the Centre for Eye Research Australia (CERA) and the University of Melbourne, set out to see if they could give them a boost.

The experiments were carried out in cultures in the lab, where brain cells were grown and then injured with a laser. A gene was then added to the mix that successfully increased protrudin levels in the cells, which in turn improved their ability to repair and regenerate.

In tests on eye and optic nerve cells, the researchers found this technique led to significant regeneration weeks after the injury. In an experiment where nerve cells from a mouse retina were grown in cell culture in a way that would normally result in widespread cell death, the technique served to offer them almost complete protection.

“What we’ve seen is the strongest regeneration of any technique we’ve used before,” says Professor Keith Martin from the Centre for Eye Research Australia. “In the past it seemed impossible we would be able to regenerate the optic nerve but this research shows the potential of gene therapy to do this.”

From here, the team plans to carry out further studies exploring how the technique can be used to protect and regenerate human retinal cells and even potentially restore sight.

The research was published in the journal Nature Communications.

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