"KAIST Develops Retinal 'Treatment' to Restore Damaged Vision"

by Jeong Ilwoong

Published 30 Mar.2025 12:29(KST)

Domestic researchers have succeeded in developing a retinal treatment that restores ‘damaged’ vision. While retinal disease treatments that inhibit disease progression have been developed in the past, the development of effective treatments capable of restoring already damaged vision had been lacking, which adds significance to this research outcome.

KAIST announced on the 30th that Professor Jinwoo Kim’s research team from the Department of Biological Sciences has developed a treatment method that can restore damaged vision through retinal nerve regeneration.

(From left) Mu-seong Kim, PhD candidate, Department of Biological Sciences, KAIST; Jin-woo Kim, Professor; Eun-jung Lee, PhD. Courtesy of KAIST

The research team administered a substance that blocks the PROX1 protein, which inhibits retinal regeneration, into the eyes of experimental mice, inducing retinal tissue nerve regeneration and vision recovery, confirming that the effect lasted for more than six months.

This study is significant as it is the world’s first case of inducing long-term nerve regeneration in the mammalian retina.

With global population aging progressing, the number of patients with retinal diseases is steadily increasing, but until now, there has been no treatment method capable of restoring the retina and vision of damaged patients. This is because damaged retinas do not regenerate.

Research on retinal regeneration in poikilothermic animals such as fish has shown that when the retina is damaged, M?ller glia cells present inside the retina dedifferentiate into neural progenitor cells and then generate new nerve cells.

In contrast, mammals including humans have lost this function, so retinal regeneration does not occur, and damage remains permanent. This is why there has been no development of treatments for retinal and vision recovery until now.

However, by discovering the PROX1 protein as a factor that inhibits the dedifferentiation of M?ller glia cells in mammals, the research team expects to offer new hope for patients with degenerative retinal diseases who previously had no treatment options.

PROX1 is a protein produced in nerve cells within neural tissues such as the retina, hippocampus, and spinal cord.

The research team found that the PROX1 protein accumulates in the M?ller glia of damaged retinas in experimental mice but does not accumulate in the M?ller glia of fish, where regeneration is active. They also proved that the PROX1 in M?ller glia is not produced internally but is secreted by surrounding nerve cells that cannot degrade it, and then taken up by the M?ller glia.

Based on this movement of the PROX1 protein, the research team developed a method to restore the nerve regeneration ability of M?ller glia by removing PROX1 extracellularly before it reaches the M?ller glia after being secreted from nerve cells.

This method uses an antibody that binds to PROX1, showing superior binding affinity compared to existing antibodies.

In particular, in the retinas of disease-model experimental mice administered with the PROX1 neutralizing antibody, nerve regeneration occurred actively, and when delivered as a gene therapy to the retinas of congenital retinal degenerative disease model mice, continuous nerve cell generation and vision recovery were maintained for more than six months.

Meanwhile, this research was conducted with support from the Korea Research Foundation’s Mid-Career Researcher Support Program and the National New Drug Development Project. The paper was completed with Dr. Eunjeong Lee from Celliaz Inc. and KAIST doctoral student Moosung Kim as co-first authors. The paper was published online on the 26th in the international academic journal Nature Communications.