Shulou(Shulou.com)11/24 Report--

CTOnews.com, May 4 (Xinhua)-- scientists have developed a new type of gene therapy that restores eye cells to a youthful state, reversing optic neuropathy caused by insufficient blood supply, a common senile eye disease that can lead to sudden blindness. This gene therapy has been successfully tested in non-human primates, opening up new possibilities for the treatment of age-related diseases.

The gene therapy was developed by Harvard Medical School and Boston biotechnology company Life Biosciences, which presented their results at the 2023 meeting of the Association for Visual and Ophthalmic Research (ARVO). Sharon Rosenzweig-Lipson, chief scientific officer of Life Bio, said: "We are pleased to be able to present these truly groundbreaking data, which further validate Life Bio's innovative approach to cell recovery."

The gene therapy works by using four genes-Oct4, Sox2, Klf4 and c-Myc-to reprogram cells to return to their embryonic state and even become stem cells. In recent years, researchers have found that they can use Yamanaka factors to locally reprogram cells to return to the period when they became adult cells, but not yet affected by adverse epigenetic markers (epigenetic marks) changes caused by aging. Epigenetic markers are chemicals attached to DNA that determine which genes in a cell are turned on ("expressed") and which are turned off. These markers change over time, so the genes we turn on when we are young may turn off with age, and vice versa, often leading to signs of aging and age-related diseases.

To test the gene therapy, the researchers used lasers to damage the eyes of 10 non-human primates to resemble ischemic optic neuropathy (NAION), an eye disease caused by an insufficient blood supply to the optic nerve, which is diagnosed each year by about 6000 Americans, most of them over the age of 50. NAION can cause sudden, severe, and often permanent blindness in the eyes. The exact cause of NAION is not known, and there is no known treatment. Six of the animals were injected with genes containing three kinds of mountain factor (OSK) and the other four were injected with a control solution using gene therapy. They designed the gene therapy so that the expression of these genes can be induced by administration of the antibiotic doxycycline-which is equivalent to giving them a switch to control the start and stop of gene therapy. They gave all animals doxycycline for five weeks, and regularly checked their retinas for response to light, using an objective test called pattern TV retinogram (pERG). They also measured the number of healthy axonal bundles in the animals' eyes.

The results showed that the animals receiving gene therapy performed significantly better than the control group on pERG tests, and there was a significant improvement in the number of healthy nerve bundles, which the researchers said indicated that vision had been restored. Bruce Ksander, co-leader of the study, said he thought the treatment could be repeated and, if necessary, could be maintained. The results of the study have not yet been published, but if they can withstand peer review, successful experiments in primates will be the best evidence to date that local reprogramming is a viable way to treat age-related diseases.

"this approach applies not only to NAION, but even to vision, and we are pleased to be able to share data that support the continued development of our scientific platform to address age-related diseases and restore human health," Rosenzweig-Lipson said.

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