UVA Scientists Develop Gene Therapy to Treat Glaucoma

Scientists may have just changed the future for millions of people facing blindness from glaucoma.

Researchers at the University of Virginia and Northwestern University developed a targeted gene therapy that reduces dangerous pressure buildup in the eye, the main cause of glaucoma. The treatment uses virus-sized particles to deliver therapy directly where it's needed, potentially replacing daily eye drops and risky surgery with a single injection.

Evan Scott, director of UVA's Institute for Nanoscale Scientific and Technological Advanced Research, led the breakthrough with collaborators Mark Johnson and Ben Thompson at Northwestern. They published their findings in the medical journal JCI Insight this month.

Glaucoma affects tens of millions of people worldwide and costs billions of dollars annually in treatments and surgeries. More than 93% of diagnoses happen in patients over 60, making it one of the leading causes of blindness as the global population ages.

Current treatments don't work well for many patients. Daily eye drops cause significant side effects, and people often forget to take them. Surgery is invasive and carries risks that some patients can't tolerate.

The team discovered a gene called Prox1 that controls pressure in the eye by regulating fluid drainage. They engineered tiny biodegradable particles that target only the specific cells containing this gene, avoiding the side effects that plague current medications.

These nanotherapeutics work because they're the same size as viruses, allowing them to interact naturally with cells and the immune system. The particles safely break down and leave the body after delivering their therapeutic payload.

The Bright Side

The research gives scientists a powerful new tool to study exactly how glaucoma develops and test different cures. The team created a mouse model that mimics the chronic nature of human glaucoma, opening doors for faster advances.

Scott's team brings together three crucial specialties. Johnson spent decades studying fluid flow in eyes. Thompson developed mouse models and identified the key gene target. Scott's lab created the delivery system that makes the therapy work without harming other parts of the eye.

The therapy is at least five years from human testing, but the goal is ambitious and life-changing: a permanent fix with one injection. For the 80 million people worldwide living with glaucoma, that single shot could mean keeping their sight for life.