Novel gene therapy shows promise for treating multiple eye diseases

Mitochondria are known as the “powerhouses” of the cell because they manage the production of energy, however researchers had previously discovered that their performance decreases in the retina of people with eye diseases. This link to a deterioration in sight led them to investigate the potential of therapies to rescue struggling mitochondria.

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.

Professor Jane Farrar, senior author of the research article and Professor in Trinity’s School of Genetics and Microbiology, said:

“Our work provides clear evidence supporting using this novel gene therapeutic approach for multiple eye disorders. It also suggests that the ophNdi1 therapeutic platform targeting mitochondrial dysfunction could have applications for other devastating conditions beyond the eye in which mitochondrial dysfunction is in play.”

Reference:

Chadderton, N.; Palfi, A.; Maloney, D.M.; Carrigan, M.; Finnegan, L.K.; Hanlon, K.S.; Shortall, C.; O’Reilly, M.; Humphries, P.; Cassidy, L.; Kenna, P.F.; Millington-Ward, S.; Farrar, G.J. Optimisation of AAV-NDI1 Significantly Enhances Its Therapeutic Value for Correcting Retinal Mitochondrial Dysfunction. Pharmaceutics 2023, 15, 322. https://doi.org/10.3390/pharmaceutics15020322