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Study shows gene therapy potential to regenerate optic nerve, Aussie professor says

A Melbourne professor has helped lead new research using a gene therapy to regenerate damaged optic nerve fibres, in a discovery that could determine future glaucoma treatments.

The pre-clinical study by Professor Keith Martin, from the Centre for Eye Research Australia (CERA) and University of Melbourne, was published in the journal Nature Communications this month and also involved Dr Richard Eva, Dr Veselina Petrova and Professor James Fawcett from the John van Geest Centre for Brain Repair at the University of Cambridge.

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.

According to CERA, the researchers used a cell culture system to grow brain cells in the lab. They 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.

Martin said 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’ve seen is the strongest regeneration of any technique we’ve used before,’’ he said.

“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.’’

The international team is continuing its collaboration in Melbourne and Cambridge. Martin said next steps were to explore the ability of protrudin to protect and regenerate human retinal cells.

The research was supported in the UK by The Medical Research Council, Fight for Sight, The Bill and Melinda Gates Foundation, Cambridge Eye Trust and the National Eye Research Council.

The open source study entitled: ‘Protrudin functions from the endoplasmic reticulum to support axon regeneration in the adult CNS’, can be found here.

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