Domestic researchers have found a way to regenerate retinas damaged by retinal diseases, inspired by fish. With the goal of entering clinical trials in 2028, it is expected to offer new hope to patients with vision loss.
A research team led by Professor Kim Jin-woo of the Korea Advanced Institute of Science and Technology (KAIST) announced on the 30th that they have developed a retinal nerve regeneration treatment method to restore damaged vision.
The number of patients at risk of vision loss due to retinal diseases is estimated to reach 300 million worldwide. Although new treatments for retinal diseases have recently been developed, offering a path to prevent vision loss, there are still no effective treatments that can restore already damaged vision.
The KAIST research team discovered the protein 'PROX1' that inhibits the dedifferentiation of mammalian Müller glial cells. PROX1 is known to be produced in nerve cells within neural tissues such as the retina, hippocampus, and spinal cord, and it inhibits the division of neural stem cells while inducing differentiation into nerve cells.
Müller glial cells are found in ectothermic organisms, such as fish, where retinal regeneration is active. These cells have the ability to dedifferentiate into neural progenitor cells and then become new nerve cells. It is known that this function has disappeared in humans and other mammals, preventing retinal regeneration.
The research team found that while PROX1 protein accumulates in Müller glial cells in damaged mouse retinas, it does not accumulate in Müller glia of fish with active regeneration. It was revealed that the PROX1 in Müller glia is not generated internally but is accepted by Müller glia from surrounding nerve cells that cannot break it down and secrete it.
The researchers found a method to remove PROX1 using antibodies that bind to it. Based on the movement of the PROX1 protein, they aimed to restore the nerve regeneration capabilities of Müller glia by removing the PROX1 secreted from nerve cells before it reaches Müller glia from outside the cell. In fact, when antibodies were injected into the eyes of mice with damaged retinas, the nerves in the retinal tissues regenerated, showing vision recovery effects. This effect lasted for over 6 months.
The retinal regeneration-inducing treatment developed by the research team is being developed by Celiaz, a startup company founded by KAIST faculty, with a target date for entering clinical trials in 2028.
Dr. Lee Eun-jung, the first author of the paper and a postdoctoral researcher, noted, “We will complete the work to improve the efficacy of the PROX1 neutralizing antibody and soon aim for efficacy and safety assessments using various animals, as well as administration to retinal disease patients,” adding, “We will conduct research to provide practical help to patients exposed to the risk of blindness without appropriate treatment.”
The research results were introduced on the 26th in the international journal 'Nature Communications.'
References
Nature Communications (2025), DOI: https://doi.org/110.1038/s41467-025-58290-8