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
Novel Investment Paradigms for Regenerative Healthcare Ecosystems
Introduction The transition toward regenerative healthcare ecosystems—anchored in wellness optimization, disease prevention, eradication strategies, and healthy longevity—necessitates a structural reconfiguration of capital architectures, governance models, and incentive design. Regenerative healthcare, by definition, transcends episodic [...]
What If Consciousness Exists Beyond Your Brain
Scientists still don’t know how consciousness emerges from the brain. New ideas suggest it may not emerge at all, but instead be a basic feature of reality. Is consciousness produced by the brain, or [...]
Scientists Discover Way To Treat Lung Cancer and Its Deadly Side Effect Together
A new approach using lipid nanoparticles to deliver genetic material is showing promise in tackling two major challenges in lung cancer at once.Researchers at Oregon State University have designed a new way to tackle two of [...]
Saunas Activate Your Immune System
A brief sauna session may quietly mobilize the immune system. A sauna session may do more than raise your heart rate and body temperature. A new study from Finland found that it also briefly [...]
Why music from your youth still has such an intense effect years later: A psychological perspective
You're driving, and suddenly a familiar song fills the air. Before you even know it, a wave of emotions comes over you – not just memories, but a deep, almost physical feeling. This powerful [...]
AI to antibody in days: breaking the wet lab bottleneck via high-throughput integration
The role of artificial intelligence (AI) in drug design has fundamentally shifted from a speculative tool to a central pillar of pharmaceutical research and development (R&D). Sino Biological plays a critical role in this [...]
Regenerative Healthcare by Design: Engineering Health-Centric Buildings and Urban Ecosystems
Introduction The next evolution of healthcare will not be confined to hospitals, clinics, or episodic interventions—it will be embedded into the infrastructure of everyday life. Regenerative health ecosystems require a systemic re-architecture of how [...]
Scientists Warn: Humanity Has Pushed the Planet Past Its Limits
Human population and consumption have surpassed Earth’s limits, increasing risks to climate and global stability. The Earth is already operating beyond its capacity to sustainably support the global population, according to new research highlighting [...]
Breakthrough Study Reveals Why Damaged Nerves Struggle To Heal
A newly identified molecular mechanism reveals how neurons weigh survival against repair after injury. Scientists at the Icahn School of Medicine at Mount Sinai have identified a molecular switch in neurons that limits the regrowth of [...]
Popular Vitamin B3 Supplements May Help Cancer Cells Survive, Scientists Warn
A new study raises important questions about widely used NAD+ supplements, suggesting that compounds often taken to boost energy and support healthy aging may have unintended consequences in cancer treatment. Millions of Americans take [...]
Scientists Discover Cancer Tumors Are “Addicted” to This Common Antioxidant
Cancer cells may be exploiting a common antioxidant as fuel, revealing a potential weakness that future therapies could target. Cancer cells may be tapping into an unexpected energy source: an antioxidant long associated with [...]
Nanotube injector transfers cytoplasmic contents and organelles between living cells safely
Cells are not isolated units; they continuously exchange proteins, genetic material, and even entire organelles with their neighbors. Intercellular transfer influences how tissues develop, respond to stress, and repair damage. In certain cancers, for [...]
CEO of America’s largest public hospital system is ready to replace radiologists with AI
The chief executive of America’s largest public hospital system says he is prepared to start replacing radiologists with artificial intelligence in some circumstances, once the regulatory landscape catches up. Mitchell H. Katz, MD, president [...]
Our books now available worldwide!
Online Sellers other than Amazon, Routledge, and IOPP Indigo Global Health Care Equivalency in the Age of Nanotechnology, Nanomedicine and Artifcial Intelligence Global Health Care Equivalency In The Age Of Nanotechnology, Nanomedicine And Artificial [...]
Study finds higher heart disease risk in long COVID patients
People with long COVID are at increased risk of developing cardiovascular disease, according to a new study from Karolinska Institutet published in eClinicalMedicine. The results show that the risk of conditions such as cardiac arrhythmias [...]
The Corona variant Cicada is here – we know that
Online and on social media, reports are piling up about a new Sars-Cov-2 variant that is currently on the rise: BA.3.2, also known as Cicada. That's what it's all about: The Omicron variant BA.3.2, [...]
