NEW GENE THERAPY COULD BE SUCCESSFUL IN TREATING AGE-RELATED EYE ILLNESSES

A GENE therapy that is already showing promise for treating one age-related eye disease could help a number of different eye problems, researchers have found.

Researchers from Trinity College Dublin have discovered that a potential new gene therapeutic approach for age-related macular degeneration may also be effective in treating patients living with other eye diseases in the future including glaucoma.

The work published in the journal Pharmaceutics details how the gene therapy boosts mitochondrial performance in retinal ganglion cells, the cells dysfunctional in diseases such as glaucoma.

More than 100,000 people in Ireland are living with age-related macular degeneration. It is the number one cause of sight loss in those over the age of 50 years here.

Glaucoma is also a major factor in sight loss in the same age group and the incidence of it is growing in Ireland with around 4% of those over the age of 50 having the condition.

The results of the latest research is consistent with those observed, and previously published by the same group, showing benefit in age related macular degeneration (AMD) models and highlights the potential value of the gene therapy — ophNdi1 —for multiple eye diseases.

‘Because a loss of retinal ganglion cells leads to sight loss in many conditions including inherited optic neuropathies and glaucoma, we are excited that this potential therapeutic approach could provide benefit to many patients in the future,’ says Dr Naomi Chadderton, first author of the research article and a scientist in Trinity’s School of Genetics and Microbiology.

‘Our study shows that ophNdi1 is protective in three models of mitochondrial dysfunction. Notably, the optimisation of the therapy, which is outlined in the study, allows for use of a lower therapeutic dose.;

Mitochondria are known as the “powerhouses” of the cell because they manage the production of energy but their performance decreases in the retina of people with eye diseases.

The gene therapy ophNdi1 uses a virus to access the cells that are suffering and deliver the code needed to give mitochondria a lifeline, enabling them to generate extra energy and continue to function in supporting vision.