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Based on years of research, the gene-therapy treatment for glaucoma developed with a biotech company can help prevent vision loss.
Scientists from Trinity College Dublin have developed a novel and promising treatment for glaucoma that is based on gene therapy.
Based in Trinity’s Smurfit Institute of Genetics, the team worked in collaboration with biotechnology company Exhaura. They developed a gene therapy-based approach that can decrease intraocular pressure in pre-clinical models of glaucoma, a group of eye conditions that can cause vision loss and blindness due to damage to the optic nerve.
Glaucoma affects around 80m people globally, and experts project an increase to more than 110m by 2040, according to Trinity. The main risk factor arises out of an increase in intraocular pressure which, when left unchecked, damages the optic nerve that sends light signals to the brain.
The pressure is caused by a build-up of unwanted proteins that block drainage channels within the eye. Over time, fluid accumulates in the eye and intraocular pressure increases.
While conventional topical eye drops help prevent disease progression, around one in 10 patients become resistant to the treatment – putting them at risk of permanent loss of vision.
Dr Jeffrey O’Callaghan, postdoctoral research fellow at Trinity and first author of the study, said that the novel approach using gene therapy is the culmination of more than seven years of research.
“We are now hopeful that this therapy will pave the way to the development of treatments for other forms of blinding eye diseases,” O’Callaghan said.
The novel treatment involves the use of a single injection of a viral vector, which is a virus the scientists have manipulated to deliver specific instructions to cells in the body. It can increase the flow of aqueous fluid from the front of the eye and thereby decrease pressure in the eye.
Scientists, ranging from geneticists and ophthalmologists to translational biologists, used multiple models of the disease to test the therapy and used donor human eyes to screen the therapeutic efficacy of the approach.
“This exciting project allowed us to bridge the gap between academia and industry, and work very closely with a gene therapy company to develop a cutting-edge therapy,” said Prof Matthew Campbell, a professor in genetics at Trinity.
“We believe [the gene therapy] holds immense promise for patients in the future.”
Findings were published this week in the international journal, Science Advances, and the study was supported by the Science Foundation Ireland, the European Research Council and Enterprise Ireland.
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