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Insight
July 2024

Researchers at Macquarie University have uncovered a promising gene therapy approach that could offer new treatment options for glaucoma, with potential benefits for other neurodegenerative diseases such as Alzheimer’s.

AI Depiction of DNA strand


The Tau protein plays a critical role in the brain and central nervous system, but when produced in excess, it can create tangles that block the normal function of cells. These tangles have been linked to Alzheimer’s disease.

Interestingly, Tau has also been identified in retinal cells, with Macquarie University’s Vision Science Group suggesting that it may contribute to glaucoma development.

In their latest research, published in Acta Neuropathologica, the team demonstrated the importance of Tau in maintaining retinal health.

Using gene therapy, the researchers manipulated the levels of Tau in retinal cells, either increasing or decreasing its expression.

Both approaches negatively impacted retinal structure and function, indicating that maintaining an optimal level of Tau is essential for eye health.

In their experimental glaucoma model, excess Tau worsened retinal degeneration, while reducing Tau to healthier levels offered significant protection against this damage.

The researchers developed a gene therapy 'switch' delivered by a viral vector, capable of crossing both the blood-brain and blood-retinal barriers.

“These results highlight the crucial role of Tau protein levels in safeguarding retinal health and underscore the potential of targeting Tau as a therapy for glaucoma,” the study authors noted.

The research team will now move on to further testing in animal models, with human clinical trials still a few years away.

This article has been republished courtesy of Insight News.