Medical Breakthrough: Seattle Man Is First Adult to Get New Gene Therapy for Vision
Toby Willis’ eyesight started deteriorating when he was just a kid, and by the time he was in his early 40s, his visual world had dimmed to shadows and silhouettes. Willis recently became the first adult to undergo the first gene therapy approved in the U.S. for treatment of an inherited disease. The medication, called Luxturna, is injected in the eye and uses a harmless virus to deliver working versions of a gene to the cells of patients whose own genes are defective. For physicians who have helplessly watched patients like Willis go blind, even an imperfect treatment represents a breakthrough. For gene-therapy researchers, it’s a thrilling validation. “I think this is a game changer,” said Dr. Timothy Cherry, who’s exploring different ways to repair inherited forms of blindness at Seattle Children’s Research Institute. “What I see as inspiring is that this shows us that the fundamental idea works.”
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