Vision is one of the most critical human senses, yet over 300 million people globally face the threat of vision loss due to retinal diseases. While current treatments can slow the decline, no therapy has been able to reverse vision loss—until now.
In a major scientific advancement, researchers at KAIST (Korea Advanced Institute of Science and Technology) have developed a revolutionary drug that successfully restores vision by regenerating damaged retinal nerves.
On March 30, KAIST announced that a team led by Professor Jin Woo Kim from the Department of Biological Sciences has created a therapy that promotes retinal nerve regeneration, offering renewed hope to those affected by degenerative eye diseases.
In tests on disease-model mice, the team achieved both retinal regeneration and vision restoration by blocking the protein PROX1 (prospero homeobox 1), a natural inhibitor of retinal repair. Remarkably, the effects of the treatment lasted more than six months, marking the first-ever successful long-term neural regeneration in the mammalian retina.
This research opens a new chapter in the fight against irreversible blindness, especially as the global population ages and cases of retinal degeneration rise. Until now, the main challenge in developing curative treatments has been the inability of mammalian retinas to regenerate after injury.
Unlike mammals, cold-blooded animals like fish have a natural ability to repair retinal damage. In these species, Müller glia cells can dedifferentiate into retinal progenitor cells that form new neurons. In mammals, however, this regenerative process is suppressed.
The KAIST study identified the PROX1 protein as a key factor preventing regeneration in mammals. PROX1 exists in the neurons of the retina, hippocampus, and spinal cord, where it limits the proliferation of neural stem cells and drives them toward differentiation.
Crucially, the team found that in damaged mouse retinas, PROX1 accumulates inside Müller glia cells, unlike in fish where it is absent. Even more surprising, this protein is not produced by the Müller glia themselves but is instead absorbed from neighboring neurons that secrete it due to a failure in degradation.
This discovery not only highlights a major biological barrier to vision recovery but also offers a precise therapeutic target for future treatments.
By turning off the signal that blocks retinal regeneration, KAIST’s breakthrough could one day help millions of people regain their sight—a feat that, until now, belonged only to science fiction.
