A clear vision for future treatment of retinal mitochondrial dysfunction

Novel gene therapy could repair malfunctioning mitochondria in retinal cells, improving vision for those suffering from glaucoma.  

Researchers from Trinity College Dublin (Ireland) have recently published an article in Pharmaceutics presenting a new gene therapy candidate that could potentially treat multiple eye diseases, such as glaucoma.

The loss of vision suffered by those with eye diseases, such as glaucoma or inherited optic neuropathies, can be attributed to the deterioration of the retinal ganglion cells. This deterioration is especially seen in the mitochondria of these cells. Mitochondria are vital to a cell as they produce ATP, which is the source of energy for cell function. The deterioration of these organelles over time leads to reduced function of the retinal ganglion cells and, ultimately, loss of vision for the patient. This led the research group to start investigating novel therapies that could prevent mitochondrial decay from occurring.

The gene therapy, termed ophNidi1, utilizes a virus to gain access to the damaged retinal ganglion cells. The virus delivers a genetic code that improves mitochondrial function in the retinal cells.

“Our study shows that ophNdi1 is protective in three models of mitochondrial dysfunction. Notably, the optimization of the therapy, which is outlined in the study, allows for use of a lower therapeutic dose,” commented Naomi Chadderton, first author of the article.

The therapy is still in its early stages so has yet to be rolled out to patients, however, the authors are highly optimistic and believe it can be applied to other mitochondrial diseases in the future.

“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,” stated Jane Farrar, senior author of the article.