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First Patients Treated With New Gene Therapy for Inherited Vision Disorders

A teen in Boston and an adult in Los Angeles have become the first non-trial patients to undergo a gene therapy treatment using a virus to deliver new genes to patients with an inherited disorder that causes loss of sight.

The medication, called Luxturna, is injected in the eye, and targets a single, rare mutation.

The Seattle Times reported over the weekend that on March 27, 13-year-old Jack Hogan became the first non-experimental patient treated with Luxturna, followed a few hours later by 44-year-old software engineer Toby Willis, a Seattle man who was treated in Los Angeles.


In the procedure, a doctor uses a needle to inject a tiny amount of viral solution behind the retina, the Times reported. Each injection contains about 150 billion viruses. The viruses invade cells with a genetic defect, depositing new genes into the nuclei of the cells with the defects.

In clinical trials, 27 out of 29 patients experienced improvements in eyesight that lasted at least a year, and more than 70 percent were able to navigate better in very low light, the Times reported. Some participants experienced dramatic revelations, like seeing the stars for the first time. One patient’s vision got worse.

“I think this is a game changer,” Timothy Cherry, who is researching treatments for inherited blindness at Seattle Children’s Research Institute told the Times. “What I see as inspiring is that this shows us that the fundamental idea works.”

READ MORE From the Seattle Times

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