Vision is one of the most crucial human senses, yet over 300 million people worldwide are at risk of vision loss due to various retinal diseases. While recent advancements in retinal disease treatments have successfully slowed disease progression, no effective therapy has been developed to restore already lost vision-until now. KAIST researchers have successfully developed a novel drug to restore vision.
KAIST (represented by President Kwang Hyung Lee) announced on the 30th of March that a research team led by Professor Jin Woo Kim from the Department of Biological Sciences has developed a treatment method that restores vision through retinal nerve regeneration.
The research team successfully induced retinal regeneration and vision recovery in a disease-model mouse by administering a compound that blocks the PROX1 (prospero homeobox 1) protein, which suppresses retinal regeneration. Furthermore, the effect lasted for more than six months.
This study marks the first successful induction of long-term neural regeneration in mammalian retinas, offering new hope to patients with degenerative retinal diseases who previously had no treatment options.
As the global population continues to age, the number of retinal disease patients is steadily increasing. However, no treatments exist to restore damaged retinas and vision. The primary reason for this is the mammalian retina’s inability to regenerate once damaged.
Studies on cold-blooded animals, such as fish-known for their robust retinal regeneration-have shown that retinal injuries trigger Müller glia cells to dedifferentiate into retinal progenitor cells, which then generate new neurons. However, in mammals, this process is impaired, leading to permanent retinal damage.
Through this study, the research team identified the PROX1 protein as a key inhibitor of Müller glia dedifferentiation in mammals. PROX1 is a protein found in neurons of the retina, hippocampus, and spinal cord, where it suppresses neural stem cell proliferation and promotes differentiation into neurons.
The researchers discovered that PROX1 accumulates in damaged mouse retinal Müller glia, but is absent in the highly regenerative Müller glia of fish. Furthermore, they demonstrated that the PROX1 found in Müller glia is not synthesized internally but rather taken up from surrounding neurons, which fail to degrade and instead secrete the protein.
Based on this finding, the team developed a method to restore Müller glia’s regenerative ability by eliminating extracellular PROX1 before it reaches these cells.
This approach involves using an antibody that binds to PROX1, developed by Celliaz Inc., a biotech startup founded by Professor Jin Woo Kim’s research lab. When administered to disease-model mouse retinas, this antibody significantly promoted neural regeneration. Additionally, when delivered, the antibody gene to the retinas of retinitis pigmentosa disease model mice, it enabled sustained retinal regeneration and vision restoration for over six months.
The retinal regeneration-inducing therapy is currently being developed by Celliaz Inc. for application in various degenerative retinal diseases that currently lack effective treatments. The company aims to begin clinical trials by 2028.
This study was co-authored by Dr. Eun Jung Lee of Celliaz Inc. and Museong Kim, a Ph.D. candidate at KAIST, as joint first authors. The findings were published online on March 26 in the international journal Nature Communications. (Paper Title: Restoration of retinal regenerative potential of Müller glia by disrupting intercellular Prox1 transfer | DOI: 10.1038/s41467-025-58290-8)
Dr. Eun Jung Lee stated, “We are about completing the optimization of the PROX1-neutralizing antibody (CLZ001) and move to preclinical studies before administering it to retinal disease patients. Our goal is to provide a solution for patients at risk of blindness who currently lack proper treatment options.”
This research was supported by research funds from Korean National Research Foundation (NRF) and the Korea Drug Development Foundation (KDDF).
News
Controlling This One Molecule Could Halt Alzheimer’s in Its Tracks
New research identifies the immune molecule STING as a driver of brain damage in Alzheimer’s. A new approach to Alzheimer’s disease has led to an exciting discovery that could help stop the devastating cognitive decline [...]
Cyborg tadpoles are helping us learn how brain development starts
How does our brain, which is capable of generating complex thoughts, actions and even self-reflection, grow out of essentially nothing? An experiment in tadpoles, in which an electronic implant was incorporated into a precursor [...]
Prime Editing: The Next Frontier in Genetic Medicine
By Dr. Chinta SidharthanReviewed by Benedette Cuffari, M.Sc. Discover how prime editing is redefining the future of medicine by offering highly precise, safe, and versatile DNA corrections, bringing hope for more effective treatments for genetic diseases [...]
Can scientists predict life longevity from a drop of blood?
Discover how a new epigenetic clock measures how fast you are really aging from just a drop of blood or saliva. A recent study published in the journal Nature Aging constructed an intrinsic capacity (IC) clock [...]
What is different about the NB.1.8.1 Covid variant?
For many of us, Covid-19 feels like a chapter we’ve closed – along with the days of PCR tests, mask mandates and daily case updates. But while life may feel back to normal, the [...]
Scientists discover single cell creatures can learn new behaviours
It was previously thought that learning behaviours only applied to animals with complex brain and nervous systems, but a new study has proven that this may also occur in individual cells. As a result, this new evidence may change how [...]
Virus which ’causes multiple organ failure’ found at popular Spanish holiday destination
British tourists planning trips to Spain have been warned after a deadly virus that can cause multiple organ failure has been detected in the country. The Foreign Office issued the alert on its dedicated website Travel [...]
Urgent health warning as dangerous new Covid virus from China triggers US outbreak
A dangerous new Covid variant from China is surging in California, health officials warn. The California Department of Public Health warned this week the highly contagious NB.1.8.1 strain has been detected in the state, making it the [...]
How the evolution of a single gene allowed the plague to adapt, prolonging the pandemics
Scientists have documented the way a single gene in the bacterium that causes bubonic plague, Yersinia pestis, allowed it to survive hundreds of years by adjusting its virulence and the length of time it [...]
Inhalable Nanovaccines: The Future of Needle-Free Immunization
The COVID-19 pandemic highlighted the need for adaptable and scalable vaccine technologies. While mRNA vaccines have improved disease prevention, most are delivered by intramuscular injection, which may not effectively prevent infections that begin at [...]
‘Stealthy’ lipid nanoparticles give mRNA vaccines a makeover
A new material developed at Cornell University could significantly improve the delivery and effectiveness of mRNA vaccines by replacing a commonly used ingredient that may trigger unwanted immune responses in some people. Thanks to [...]
You could be inhaling nearly 70,000 plastic particles annually, what it means for your health
Invisible plastics in the air are infiltrating our bodies and cities. Scientists reveal the urgent health dangers and outline bold solutions for a cleaner, safer future. In a recent review article published in the [...]
Experts explain how H5 avian influenza adapts to infect more animals
A new global review reveals how rapidly evolving H5 bird flu viruses are reaching new species, including dairy cattle, and stresses the urgent need for coordinated action to prevent the next pandemic. Since its [...]
3D-printed device enables precise modeling of complex human tissues in the lab
A new, easily adopted, 3D-printed device will enable scientists to create models of human tissue with even greater control and complexity. An interdisciplinary group of researchers at the University of Washington and UW Medicine [...]
Ancient DNA sheds light on evolution of relapsing fever bacteria
Researchers at the Francis Crick Institute and UCL have analyzed ancient DNA from Borrelia recurrentis, a type of bacteria that causes relapsing fever, pinpointing when it evolved to spread through lice rather than ticks, and [...]
Cold Sore Virus Linked to Alzheimer’s, Antivirals May Lower Risk
Summary: A large study suggests that symptomatic infection with herpes simplex virus 1 (HSV-1)—best known for causing cold sores—may significantly raise the risk of developing Alzheimer’s disease. Researchers found that people with HSV-1 were 80% [...]
