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.

“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 available at mivision.com.au.