Researchers at WVU Develop Promising Gene Therapy for Inherited Blindness

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Researchers at West Virginia University (WVU) are making significant strides in developing a gene therapy treatment that could restore sight for individuals with inherited blindness. Led by Dr. Visvanathan Ramamurthy, the team has received a $1.4 million grant from the National Eye Institute to focus on mutations in the PROM1 gene, a common cause of vision impairment. Their groundbreaking approach involves directly delivering functional genetic material via a harmless viral vector into the eye, with early results in mouse models showing promise for long-lasting effects.

Why it matters

Inherited blindness caused by PROM1 gene mutations affects countless individuals, yet there are currently no effective treatments available. This research at WVU represents a major breakthrough that could transform the lives of those living with this debilitating condition. If successful, the therapy could halt or even reverse vision loss, providing hope to patients and their families.

The details

The WVU research team, which includes collaborators like Wen Tao Deng and Peter Stoilov, is building on nearly two decades of work in the field of inherited blindness. Their gene therapy approach involves directly delivering functional genetic material via a harmless viral vector into the eye, with early results in mouse models showing the potential for long-lasting effects of at least one year. The researchers are also investigating whether the therapy can be effective in later stages of the disease, as most patients don't seek treatment until vision loss has already progressed.

• The WVU Eye Institute is planning an expansion that will provide the infrastructure needed for human clinical trials by late 2027.
• The research team received a $1.4 million grant from the National Eye Institute in 2026 to fund their work.

What they’re saying

What’s next

The WVU Eye Institute is planning an expansion that will provide the infrastructure needed for human clinical trials of the gene therapy treatment by late 2027.