Newly Funded Research Promoting Retinal Cell Axon Regeneration is Underway Thanks to NEI’s Audacious Goals Initiative

Optic nerve regeneration could be the answer to curing glaucoma, or at least substantially slowing the progression of the disease.

Thanks to funding from the National Eye Institute (NEI) Audacious Goals Initiative, there are currently six truly audacious studies being done in the area of optic nerve regeneration.

One of the most interesting and promising studies is from Yale University, where researchers are working to find genes that contribute to the regeneration of axons. Axons are long fibers that grow from nerve cells to transmit electrical impulses, in this case from the sensory nerve cells in the retina, up the optic nerve, and into the brain. This exciting work, underway right now, will lay the foundation for gene therapies aimed at restoring sight by healing optic nerve damage seen in glaucoma patients.

Sifting Through Genes: Phase 1

The research team, led by Yale’s Stephen M. Strittmatter, began their search in September with a veritable mountain of possible candidate genes, over 17,000. They weeded through this wide selection, eventually narrowing it down to the most promising 450 genes. The rest of the study will be taking place over the coming months, with researchers beginning trials using an optic nerve injury model.

As the experiment progresses, the selected genes will be tested to see if any of them induce regeneration of the damaged nerve. Positive results will then be verified using a species of roundworm called C. elegans—the first multicellular organism to have its whole genome sequenced and a well-established model organism for neural development studies. Researchers will see whether the positive genes are active in the worm, which would indicate that the gene functions across species. Based on those results, the strongest candidate genes can then be analyzed in greater detail in order to better understand how they work. Since many species share genetic similarities, this strategy is promising.

Audacious Goals and Future Studies

Next step studies like these are vital to continued progress in combatting glaucoma-related blindness. Yale’s axon regeneration research is one of six new projects funded by the National Eye Institute (NEI) Audacious Goals Initiative, which has targeted $12.4 million of funding over the next three years aimed at restoring vision in patients with damaged retinas. “Understanding factors that mediate the regeneration of neurons and the growth of axons is crucial for the development of breakthrough therapies for blinding diseases. What we learn through these projects will have a health impact beyond vision,” said director of NEI Paul Sieving.

The other projects making up this round of the Audacious Goals Initiative, which was announced on September 1, 2016, are built on recent progress made in the area of neural regeneration, and run the gamut from RNA sequencing to mass spectrometry to DNA modification. These are ambitious and innovative studies that will hopefully be instrumental in achieving what was previously thought to be impossible: restoring sight to damaged eyes.

None of it would have been possible without funding from the NEI’s Audacious Goals Initiative These are important first steps but there is a long road ahead and much more research to be done before work based on these studies can lead to effective treatments for optic nerve injuries.


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First posted December 23, 2016; Reviewed June 16, 2022