Macquarie University researchers have identified a gene therapy that could help treat glaucoma, with potential applications for other neurodegenerative conditions such as Alzheimer’s disease.
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.
The protein has also been found in retinal cells, with research by Macquarie University’s Vision Science Group finding that it also likely plays a role in glaucoma.
In the latest study by the group, published in Acta Neuropathologica, the team found that Tau played a role in retinal integrity.
The researchers employed gene therapy for Tau where they both over- and under-expressed the protein.
Both manipulations were found to adversely affect retinal structural and functional measures, therefore suggesting that there is an ideal Tau level promote optimal retinal health.
In the experimental glaucoma model, Tau over-expression increased inner retinal degeneration, while Tau silencing to healthy levels provided significant protection against these degenerative changes.
The ‘switch’ the team developed is a gene therapy carried by a viral vector, which has the unique ability to cross both the hard-to-penetrate blood-brain and blood retinal barriers.
“These findings underscore the critical role of endogenous Tau protein levels in preserving retinal integrity and emphasise the therapeutic potential of targeting Tau in glaucoma pathology,” the study authors said.
The next step will involve further testing in animal models, with any human clinical trials still several years in the future.
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