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Breakthrough in Gene Therapy Brings New Hope to Glaucoma Patients

26 Jul 2024
Admin
News Article

Breakthrough in Gene Therapy Brings New Hope to Glaucoma Patients

Overview

Macquarie University researchers have identified a gene therapy that could help treat the eye disease glaucoma, with potential applications for other neurodegenerative conditions such as Alzheimer’s disease.

Long term Impacts of Glaucoma

Glaucoma may result in irreversible blindness over 10 to 15 years, if left untreated. Like Alzheimer’s, it becomes more common with advancing age.
In India, 11.2 million people aged 40 and above have glaucoma, while the global cases are projected to hit 111.8 million by 2040.

Causes

Glaucoma can be caused by an imbalance in tau protein in the human body.
The protein tau is essential to the function of cells in the brain and central nervous system, but when over-produced under certain conditions, it forms tangles that clog the cells’ internal structures.
These tangles have also been found in Alzheimer's disease patients.
Tau is also present in neuronal cells in the retina, and research led by Macquarie University’s Vision Science Group has found altered tau is likely to have a significant role in the development of glaucoma.

From the Macquarie University

We have shown that tau is vital to maintaining retinal integrity, but it is a delicate balance,” says Research leader, Vivek GuptaAssociate, Associate Professor, Macquarie University.
When we over-produced tau, we observed inner retinal degeneration, but when there was too little tau, that was also detrimental. Switching off the over-production and knocking down the tau to healthy levels provided protection against the degenerative changes associated with glaucoma. This protective effect was evident in both the structural preservation of retina cells and their function. While it was not able to restore lost vision, it did stop the retinal degeneration from worsening.
He further added, “These findings highlight the critical role of tau protein in retinal health, and suggest that targeting tau could be a promising therapeutic strategy for glaucoma, particularly when administered early.

From the Lead Author

The paper’s lead author, Dr Kanishka Pushpita Maha Thananthirige, says, the team’s aim is to develop a gene therapy to use in conjunction with treatments that lower interocular pressure after glaucoma is diagnosed.
This kind of gene therapy that controls tau production can be designed to target neurons in the brain and central nervous system, as well as those in the retina. With altered, or pathogenic, tau often present in the neurons of patients with Alzheimer’s disease, there is good potential that it could also be beneficial in treating these and other neurodegenerative diseases.

The next step will involve further testing in animal models, with any human clinical trials still several years in the future.

About the Experts Involed in Trial

Associate Professor Vivek Gupta is a neurobiologist and vision scientist at Macquarie Medical School.
Dr Kanishka Pushpitha Maha Thananthirige is a vision neuroscientist at Macquarie Medical School.
Professor Stuart Graham is a glaucoma specialist and Head of Ophthalmology at Macquarie Medical School.

About Macquarie University

Founded in 1964, Macquarie University celebrates 60 years of strong tradition in pioneering thinking, ingenuity and exploration.
We continue to break new ground as we work to solve some of the world’s most pressing issues to build a positive future for all.
Ranked among the top 130 universities in the world (QS and Times Higher Ed Rankings 2024), the University produces highly sought-after graduates.
In the QS World University Rankings by subject in 2024, the University was ranked among the Top 50 in the world for Data Science and Artificial Intelligence, Philosophy and Linguistics.