A United States research team is studying how to restore vision in people who develop a form of inherited blindness, by delivering functional genetic material via a harmless viral vector injected into the back of the eye.
The team from West Virginia University is focusing on mutations in the Prominin1, or PROM1 gene, which encodes a protein expressed throughout the body.1 While approximately 50 distinct PROM1 mutations have been identified as drivers of vision loss, a significant therapeutic gap remains.
Professor Visvanathan Ramamurthy, who is leading the study, which is supported by a three-year US$1.4 million grant from the National Eye Institute, said the team is using a mouse model of the disease to deliver functional genetic material via a harmless viral vector injected into the back of the eye.
Their findings to date indicate that a single injection can preserve or even restore vision for at least a year.
Their findings to date indicate that a single injection can preserve or even restore vision for at least a year.
“That one-year window in mice is significant,” Prof Ramamurthy explained. “It suggests the potential for a much longer period of effectiveness when translated to human patients.”
Beyond initial restoration, the team is investigating whether this therapy remains effective once the disease has progressed, a critical factor for clinical application.
“Most patients do not seek treatment at the earliest stage of the disease,” Prof Ramamurthy said. “We want to learn if therapy can still provide a benefit in the middle or even later stages of vision loss.”
Reference
- West Virginia University ENews, WVU research team working to restore sight lost to genetic eye disease, 23 Jan 2026, available at: enews.wvu.edu/articles/2026/01/23/wvu-research-team-working-to-restore-sight-lost-to-genetic-eye-disease [accessed Jan 2026].
