2018

Medical Breakthrough: Seattle Man Is First Adult to Get New Gene Therapy for Vision

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.”

About Seattle Children’s

Seattle Children’s mission is to provide hope, care and cures to help every child live the healthiest and most fulfilling life possible. Together, Seattle Children’s Hospital, Research Institute and Foundation deliver superior patient care, identify new discoveries and treatments through pediatric research, and raise funds to create better futures for patients.

Ranked as one of the top children’s hospitals in the country by U.S. News & World Report, Seattle Children’s serves as the pediatric and adolescent academic medical center for Washington, Alaska, Montana and Idaho – the largest region of any children’s hospital in the country. As one of the nation’s top five pediatric research centers, Seattle Children’s Research Institute is internationally recognized for its work in neurosciences, immunology, cancer, infectious disease, injury prevention and much more. Seattle Children’s Hospital and Research Foundation works with the Seattle Children’s Guild Association, the largest all-volunteer fundraising network for any hospital in the country, to gather community support and raise funds for uncompensated care and research. Join Seattle Children’s bold initiative – It Starts With Yes: The Campaign for Seattle Children’s – to transform children’s health for generations to come.

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