Analysis of proteins identifies new drug targets for treating inherited retinal degenerations

An international team of researchers has identified new drug targets for therapies that could benefit patients with different forms of retinitis pigmentosa and other inherited retinal diseases. Using advanced proteomics techniques, they unveiled shared critical pathways in retinitis pigmentosa disease models.

The research represents significant progress in understanding how the proteome may change in different retinal dystrophies. Published in Molecular & Cellular Proteomics, the study was carried out by researchers from the University of Eastern Finland (UEF), the University of California, Irvine, and the University of Ottawa.

Retinitis pigmentosa (RP), a group of genetic disorders that cause progressive vision loss, has long been a challenge to treat due to its genetic diversity. However, the new study led by researchers at UEF suggests that disease-modifying treatments that could benefit patients with all forms of the disease, regardless of the underlying mutation, are achievable.

The researchers demonstrated that shared pathological processes occur downstream of the initial rod cell degeneration in distinct forms of RP, opening possibilities for broad-spectrum therapeutic interventions.

This cross-institutional, multi-methodological study provided a comprehensive analysis of retinal proteins, comparing three mouse models of inherited retinal degeneration to healthy wild-type mice.

“Our data facilitate the development of new therapeutic strategies that do not rely on specific genetic mutations, potentially offering hope to millions of patients affected by retinal degenerative diseases,” says Dr. Henri Leinonen, the senior author of the study.

“We identified key retinal proteins and pathways that could be targeted to mitigate the progression of retinal degeneration,” continues Dr. Ahmed Montaser, the study’s first author and a postdoctoral researcher at the Leinonen Retina Laboratory, University of Eastern Finland, School of Pharmacy.

“Additionally, we are sharing this detailed profile of retinal proteins in both healthy and diseased states with the scientific community to encourage further research and accelerate the development of effective treatments.”