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University of Oregon researchers develop gene therapy for eye disease

The therapy could eventually provide an alternative treatment for Fuchs’ dystrophy, an eye disease that is the leading cause of corneal transplant surgery
August 3, 2021

Researchers in the Phil and Penny Knight Campus for Accelerating Scientific Impact at the University of Oregon have developed, in mice, a new gene therapy that could eventually provide an alternative treatment for Fuchs’ endothelial corneal dystrophy, a genetic eye disease affecting roughly one in 2,000 people globally. Currently, the only treatment is corneal transplant, a major surgery with associated risks and potential complications.

“When you do a transplant you make a huge difference for that person, but it's a big deal for the patient with lots of visits, lots of eye drops, lots of co-pays, and if you had a medical treatment that did not require surgery, that would be great,” said Bala Ambati, a research professor in the Knight Campus and corneal surgeon who led an eight-year study involving the development of the gene therapy. “Not only could it help patients who need a transplant, but it could also help a lot of other people who could have used that (corneal) tissue."