As one of the world’s leading causes of blindness, glaucoma destroys retinal ganglion cells (RGCs), a major part of your eye’s optic nerve cells that capture light and send visual input to your brain. In adults, RGCs have little ability to regenerate, making vision loss from glaucoma irreversible, and blindness permanent. 

Recently, however, a team of Harvard University scientists successfully reversed glaucoma-related vision loss, along with normal age-related vision loss, in mice. The study findings, published in the journal Nature, bring new hope for glaucoma patients worldwide. 

What They Did

Using gene therapy, the research team epigenetically reprogrammed eye tissue cells in mice in an effort to reverse vision loss from glaucoma, as well as normal vision loss from aging, thus restoring youthful gene function. Epigenetic therapy chemically alters gene expression without actually changing the DNA sequence. 

Only three out of the four transcription factors (the genes responsible for reprogramming cells for more youthful function – Myc, Oct3/4, Sox2, and Klf4) were used. The Harvard scientists purposely omitted Myc, as it is known to facilitate tumor growth, contribute to a wide variety of human cancers and decrease longevity, all of which are common risks of gene editing.

What They Found

Results of the experiments were dramatic, with multifactorial benefits. In the glaucomatous mice the epigenetic reprogramming supported nerve regeneration, and reversed vision loss caused by injury to the optic nerve. In non-glaucomatous mice, the gene therapy reversed age-related vision loss.

What it Means

This study is the first to demonstrate the possibility of safely reprogramming complex tissues, such as nerve cells, to an earlier, healthier degree of function.

These encouraging results will need to be repeated in different animal models before advancement to clinical trials, but this early triumph opens doors to new potential treatments and damage reversal not only regarding glaucoma, but also many other diseases, injuries and age-related conditions across body systems.

*BrightFocus Foundation. (2021, Apr. 1). A Revolutionary Approach to Restoring Vision Loss from Glaucoma.  https://www.brightfocus.org/glaucoma/article/revolutionary-approach-restoring-vision-loss-glaucoma