Advancing gene therapy for new glaucoma treatments

A research team led by CERA Managing Director Professor Keith Martin has used gene therapy to regenerate damaged optic nerve cells in a pre-clinical study, raising hopes for future glaucoma treatments.


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When a patient is diagnosed with glaucoma, their treatment will focus on slowing down or stopping the disease to prevent vision loss.
Glaucoma causes progressive damage to the optic nerve, the vital transport system which sends visual information from the eye to the brain.
“If glaucoma is left untreated or detected late it can lead to irreversible vision loss,’’ explains CERA Managing Director and world-renowned glaucoma researcher Professor Keith Martin.
“Currently, most glaucoma treatment focuses on reducing eye pressure to prevent damage to the optic nerve.
“But these treatments don’t work for about 15 per cent of patients and there is nothing we can do to repair the damage to the optic nerve once it has occurred – or to restore vision that has been lost.’’

New promise

However, promising new research has moved scientists one step closer to developing treatments which could in the future restore lost vision.
In a pre-clinical study published in Nature Communications, Professor Martin and his research partners used gene therapy to regenerate damaged optic nerve cells and prevent them from dying after injury.
Professor Martin, who led the study with Dr Richard Eva, Dr Veselina Petrova and Professor James Fawcett from the John van Geest Centre for Brain Repair at the University of Cambridge, says the findings are a potential game-changer for the treatment of glaucoma.
“It raises real hope of future treatments which could restore damage to the optic nerve and potentially even restore sight,’’ says Professor Martin.

About the research

The team tested whether a gene responsible for producing a protein known as protrudin could stimulate the regeneration of nerve cells and stop them from dying when they were injured.
They used a cell culture system to grow brain cells in the lab and then injured them using a laser before introducing a gene to increase the amount of protrudin in the cells, vastly increasing their ability to repair and regenerate.
Tests of eye and optic nerve cells found the protein enabled significant regeneration weeks after a crush injury to the optic nerve.
The research demonstrated almost complete protection of nerve cells from a mouse retina growing in cell culture, a technique which would usually be expected to result in extensive cell death.

“What we have seen in this study is by far the most powerful of any of the regeneration techniques we have tried.’’ – Professor Keith Martin
Powerful technique

Professor Martin says although the research is still in pre-clinical stage and more work needs to be done, early results are extremely encouraging.
“What we have seen in this study is by far the most powerful of any of the regeneration techniques we have tried,’’ says Professor Martin.
The international team is continuing its collaboration in Melbourne and Cambridge, and Professor Martin says future research will test the ability of protrudin to protect and regenerate human retinal cells.
Ultimately, the team hope to test the technique in clinical trial – although this is still several years away.
The international research was supported in the UK by the Medical Research Council, Fight for Sight, the Bill and Melinda Gates Foundation, Cambridge Eye Trust and National Eye Research Council.


Read the full study

Petrova, V et al. Protrudin functions from the endoplasmic reticulum to support axon regeneration in the adult CNS. Nat Comms; 5 Nov 2020; doi: 10.1038/s41467-020-19436-y


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