Dong Feng Chen, MD, PhD

Ajay Ashok, PhD

Abnormal mitochondrial function can drive damage in retinal ganglion cells (RGCs), which carry signals from the eyes to the brain. As such, mitochondrial dysfunction is increasingly being recognized as a central driver of neuronal injury, aging and irreversible blindness in diseases such as glaucoma and inherited mitochondrial optic neuropathies.

Prior research has shown that mitotherapy—the transplantation of healthy mitochondria into damaged cells—can restore normal energy production and promote recovery in models of brain and heart injury.

In a recent study led by Ajay Ashok, PhD, and supervised by Dong Feng Chen, MD, PhD, both of the Department of Ophthalmology at Schepens Eye Research Institute of Mass Eye and Ear, researchers examined whether mitotherapy could improve RGC health and encourage nerve regeneration after injury.

The team found that cultured neurons subjected to stress could take up externally delivered healthy mitochondria, leading to enhanced energy production, lowered oxidative damage, improved neuronal survival and stimulated nerve growth. The investigators then delivered mitochondria into mouse models of optic nerve damage via eye injection and showed that the mitochondria successfully integrated into RGCs and protected them from injury. This, in turn, promoted axon regrowth and functional recovery.

Together, these results suggest that mitotherapy could be a promising treatment for neurodegenerative eye diseases and other conditions involving mitochondrial failure. More broadly, because mitochondrial dysfunction is considered a hallmark of aging, this work raises the exciting possibility that restoring mitochondrial health by mitochondrial transplantation could help reverse age-related neuronal decline and promote tissue rejuvenation across neurodegenerative diseases.

Published in Free Radical Biology and Medicine on April 6, 2026 | Read the paper: “Mitochondria transplantation preserves retinal ganglion cells and promotes CNS axonal regeneration”

Summary reviewed by: Ajay Ashok, PhD, lead author, and Dong Feng Chen, MD, PhD, senior author