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Gene therapies could transform the treatment of sickle cell disease


Manny Johnson keeps catching himself rubbing a spot on the upper right side of his chest. Starting when he was a teenager, the 21-year-old from Boston depended on a port implanted there to ease his monthly blood transfusions for sickle cell disease, a genetic disorder that caused a stroke when he was three.

But in November, six months after Johnson became the first patient to receive an experimental therapy aimed at curing his disease, the port that had become part of him — requiring special approval to play sports, used when he was excused from school for a day or two every month for treatments — was removed. Johnson hasn’t needed a transfusion or had any symptoms since May.

“It’s like, wow. I’m fascinated by it,” said Johnson, who added that he almost didn’t know what to make of it when his medical team told him they wouldn’t need to see him for three whole months. “I’m so used to being next to them. It’s like: freedom.”



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