09/20/2026
💛 A promising new direction in retinal regeneration research
For years, scientists have been fascinated by something zebrafish can do that humans cannot: regenerate damaged retinal neurons.
When a zebrafish retina is injured, support cells called Müller glia can reprogram themselves, produce new retinal neurons, and help repair the retina. Mammalian Müller glia normally don’t respond this way.
Researchers at KAIST in South Korea may have discovered an important reason why.
In a study published in Nature Communications in March 2025, researchers identified a protein called PROX1 that appears to act as a brake on this regenerative process.
The surprising part? Müller glia don’t appear to make the PROX1 that accumulates inside them. Instead, the protein is transferred from neighboring retinal neurons.
When researchers interfered with this process in mice, Müller glia began reprogramming into retinal progenitor cells capable of generating new retinal neurons.
Even more encouraging for the retinitis pigmentosa community, the researchers tested the approach in a mouse model of RP. Blocking PROX1 was associated with retinal regeneration and delayed vision loss, with evidence of improved retinal function.
That does not mean we have a new treatment for people with RP yet.
This remains preclinical research. The regeneration demonstrated so far has been in animal models, and many questions must be answered about safety, effectiveness, durability, and whether the same process can restore meaningful vision in people.
But there is an exciting reason we’re watching this research.
Unlike treatments designed to correct one particular RP-causing gene, stimulating the retina’s own regenerative ability could potentially become a gene-agnostic strategy—one that might someday benefit people with many different genetic forms of retinal degeneration.
The therapy being developed from this research, currently known as CLZ001, is still in preclinical development. Human clinical trials have been discussed as a future goal, but this treatment is not currently proven in people with RP.
For families living with inherited retinal disease, discoveries like this give us another reason to keep pushing research forward.
We don’t know yet whether unlocking the regenerative potential of Müller glia will ultimately restore vision in humans.
But scientists are beginning to understand why the mammalian retina doesn’t regenerate—and how that biological barrier might potentially be overcome.
That is a development worth watching. 💛
— RP Gene Research Foundation
Our mission is to raise awareness and funds for research into retinitis pigmentosa, including PRPF31 and gene-agnostic approaches that could benefit people across many forms of inherited retinal disease.
