Novel gene therapy shows promise in reversing vision loss in primates

The team's goal is to create a potential treatment for a condition known as non-arteritic anterior ischemic optic neuropathy (NAION).
Mrigakshi Dixit
Representational image
Representational image


Our eyes become more vulnerable to vision-related disorders as we age. 

Now, in major development, a novel gene-therapy treatment has shown promise in reversing vision loss in primates. This new study lays the groundwork for the future development of human eye age-related treatments. 

The results of preclinical trials led by scientists from Harvard Medical School and the biotech company Life Biosciences were announced last month. 

The challenge? The team's goal is to create a potential treatment for a condition known as non-arteritic anterior ischemic optic neuropathy (NAION). This disorder causes a painless but sudden loss of vision in one eye due to the lack of blood flow to the optic nerve. 

Researchers aren't sure what causes NAION, but it is often characterized as an eye stroke. "NAION is the most common cause of acute optic neuropathy in people over 50, but currently has no effective treatment," said Bruce Ksander, study co-lead and associate professor of ophthalmology at Harvard, in an official press release

The development of gene therapy 

According to the official statement, this gene therapy restores visual function through a "partial epigenetic reprogramming" process. The old cells are partially reprogrammed to their younger state in this medical technique. 

For this study, the epigenome of older animals was reprogrammed through the expression of three Yamanaka factors, Oct4, Sox2, and Klf4, known collectively as OSK. 

The primates were then given an "intravitreal (in-eye) injection of doxycycline-inducible OSK viruses" to partially reprogram eye cells, just a day after the laser-induced damage to their eyes. A total of ten primates were chosen for this study, with six of them receiving the new gene therapy treatment for more than five weeks. 

The results demonstrated that the therapy was effective in restoring visual function in primate models. Furthermore, the health of their eyes' nerve fibers showed signs of improvement, indicating vision restoration. 

This ground-breaking therapy could pave the way for the development of new treatments for eye cellular rejuvenation as well as other eye-related disorders

“Demonstrating rejuvenation in nonhuman primates is a major step forward in advancing cellular rejuvenation as a way of treating both common and rare diseases in the eye and potentially other tissues. What we’ve learned in NHPs has important ramifications for research on reversing aging and is likely to be highly translational to humans. This data moves us an important step closer to the first clinical trials of how cellular rejuvenation technology could treat aging-related diseases,” said Dr. Sinclair, Co-Founder of Life Bio, and co-author of the study. 

The results of the study trial were presented at the Association for Research in Vision and Ophthalmology (ARVO) 2023 conference in New Orleans. The preliminary findings have not yet been peer-reviewed or published in a journal. 

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