Novel gene therapy has been developed as a treatment option for glaucoma sufferers thanks to a collaboration between Exhaura Limited and Trinity College Dublin. About 80 million individuals throughout the world suffer from glaucoma, an eye illness that can cause permanent blindness if left untreated.

Authorities have given multiple medications developed due to gene therapy research the green light in recent years. Although this is progress, the majority of licensed medications have only addressed extremely rare genetic disorders. In contrast, Trinity College scientists have spent the last few years developing a gene therapy to treat glaucoma, a widespread illness.

What is glaucoma?

Glaucoma is a group of eye disorders that can result in blindness. Even though there are different types of glaucoma, in all cases, increased pressure inside the eye damages the optical nerve, which leads to vision loss.

The optical nerve carries visual impulses from the retina to the brain. However, in glaucoma, drainage pathways become blocked due to the accumulation of undesirable proteins inside the eye. The pressure from the buildup of fluid inside the eye causes damage to the optic nerve.

How can gene therapy help glaucoma patients?

Glaucoma sufferers currently utilize eye drops topically to slow the progression of the disease. Some individuals, however, eventually become resistant to the medication.

In order to conduct the study, researchers at Trinity College’s Smurfit Institute of Genetics infected eye cells with a virus.

The virus was genetically modified to incorporate a gene that produces metalloproteinase-3, or MMP-3. The enzyme is a matrix protein that increases the flow of aqueous fluid from the front of the eye, lowering internal pressure.

To assess the efficiency of the medication, the researchers conducted trials in multiple disease models as well as donor human eyes. Because the research was conducted in collaboration with industry, all experimental outputs were aimed at turning the findings into a regulated clinical program.

Matthew Campell, professor in Genetics at Trinity says: “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 that we believe holds immense promise for patients in the future.”

The team is eager to apply this method to treat further eye problems. 

Source study: Science Advances— Metric metalloproteinase-3 (MMP-3)—mediated gene therapy for glaucoma