Glaucoma leads to a shortage of energy in the cells of the retina, impairing their ability to function properly. This gene therapy works to address that issue by boosting the energy production within these cells—a common challenge in multiple eye conditions including age-related macular degeneration (AMD). At the core of this process are the mitochondria, often referred to as the “powerhouses” of the cell, which are responsible for generating the energy necessary for our cells to function.
The mitochondria manage the production of energy in our cells, however their performance declines in Glaucoma and other eye conditions. This is linked to a deterioration in vision. The AAV-NDI1 therapy uses a safe virus called adeno-assosicated virus (AAV) to deliver a code (the NDI1 gene) to cells of the retina. This helps to promote the energy production of the mitochondria and reduce the damaging effects, helping to preserve vision.
First author of the published research article, Dr Sophia Millington-Ward, Research Fellow in Trinity’s School of Genetics and Microbiology, said:
Glaucoma is a complex group of optic neuropathies and a leading cause of blindness. In Europe, roughly 1 in 30 people aged between 40 and 80 years have glaucoma, and that rises to 1 in 10 in persons over 90, so this is a really common condition that badly needs new treatment options.
It is a multifactorial condition with many different risk factors, which adds to the complexity of treating it. Current glaucoma treatments focus on the use of topical eye drops, surgery, or laser therapy, however the outcomes are variable, with some patients not responding and/or suffering serious side effects.
The need for better treatment options has inspired and motivated us to continue developing gene therapies, and we are delighted it the promise it is showing. – Dr Sophia Millington-Ward
Overall, this study highlights the potential benefit of this gene-independent therapy as a future treatment for people living with Glaucoma as well as other eye conditions.
Professor Jane Farrar, principal investigator of this study at Trinity, added:
Developing broadly applicable gene therapies for large numbers of patients is particularly important, given high development costs associated with each therapy – and here we have highlighted this therapy has real potential for boosting mitochondrial function in glaucoma. – Prof. Jane Farrar
The Research team in Trinity is funded by Science Foundation Ireland, Enterprise Ireland, Health Research Board Ireland, EU Marie Curie Innovative Training Network, and Fighting Blindness Ireland—Health Research Charities Ireland.