Scientists from Trinity College Dublin have developed a new gene therapy that could transform the treatment of glaucoma, a leading cause of blindness worldwide. This innovative therapy has been shown to protect retinal cells and improve their function, offering a significant step toward better treatment options for millions of people affected by the condition.

Glaucoma currently impacts around 80 million people globally, with existing treatments like eye drops, surgery, and laser therapy offering varying success and sometimes causing serious side effects. The new gene therapy, developed by the Trinity team, uses an approved virus to deliver an enhanced gene (eNdi1) aimed at boosting mitochondrial activity, which is critical for energy production within cells. The therapy also helps reduce harmful reactive oxygen species, which can damage cells.

In their latest study, published in the International Journal of Molecular Genetics, researchers demonstrated the effectiveness of this gene therapy in protecting retinal ganglion cells (RGCs) in an animal model of glaucoma. These cells play a crucial role in vision. Additionally, when applied to human cells derived from individuals with glaucoma, the gene therapy was found to improve oxygen consumption and energy production, indicating enhanced cellular performance.

Dr. Sophia Millington-Ward, a lead researcher on the project, highlighted the urgent need for new treatments, given the complexity of glaucoma and the variable success of current options. The promising results of this therapy have inspired further development, with the ultimate goal of translating these findings into clinical treatments.

The research team, led by senior author Professor Jane Farrar, has founded Vzarii Therapeutics to advance this gene therapy toward human clinical trials, aiming to bring this potential solution to patients suffering from glaucoma and other conditions such as dry age-related macular degeneration (AMD).

This breakthrough highlights the potential of gene therapy as a powerful tool for treating complex eye diseases and restoring vision. As the team moves toward clinical trials, the future looks brighter for individuals battling glaucoma.