Researchers at the Institute of Molecular and Clinical Ophthalmology Basel report that a high-throughput screen of more than 2,700 compounds in lab-grown human retinal organoids identified molecules that improved survival of cone photoreceptors—cells essential for sharp, color vision. The team linked the protective effect to inhibiting casein kinase 1 and says the results were also supported in a mouse model of retinal degeneration.
A research team led by Botond Roska at the Institute of Molecular and Clinical Ophthalmology Basel (IOB) has identified compounds and pathways that may help protect cone photoreceptors—retinal cells needed for reading, recognizing faces and seeing color—from degeneration.
Screening thousands of compounds in human retinal models
To search for potential protective molecules, the researchers tested more than 2,700 compounds across about 20,000 human retinal organoids, lab-grown tissue models designed to mimic key features of the human retina. The team selectively labeled cone cells, allowing them to track cone survival over time while exposing the organoids to controlled stress conditions intended to mimic disease-related injury.
The screening results pointed to both promise and risk. Some classes of compounds were associated with damage to cone cells, underscoring the need to assess retinal toxicity alongside potential benefits. Other molecules improved cone survival in the organoid system.
Casein kinase 1 emerges as a protective target
Across different stress conditions, the analysis consistently highlighted casein kinase 1 (CK1) as a mechanism linked to cone protection. The researchers reported that two kinase inhibitors showed repeated protective effects on cone survival in the organoids. According to the research summary, those benefits were also confirmed in a mouse model of retinal degeneration, suggesting the mechanism may extend beyond the organoid system.
Publication and data release
The findings were published in Neuron in a paper led by first authors Stefan E. Spirig and Álvaro Herrero-Navarro. The team also released a dataset describing the tested compounds, their molecular targets and their measured effects on cone survival, with the goal of supporting future work on therapies aimed at preserving central vision and improving systematic evaluation of retinal safety.
While the results identify drug-like molecules and a biological target associated with improved cone survival in experimental models, the work does not establish a clinical treatment and would require further validation before any potential use in patients.
