Gene therapy has helped a 9-year-old boy regain enough muscle strength to run. If successful in others, the treatment could change the lives of thousands of children with Duchenne muscular dystrophy.
The group knew that Samulski didn't have much business experience, Howell says."But, on the other hand, he knew a tremendous lot about viruses and how they work and how they might really be effectively brought into the clinical practice.""I remember Jude saying we probably won't get funding because gene therapy is an unproven technology and there are a lot of naysayers," saysToday, AskBio occupies a gleaming new headquarters in Research Triangle, N.C.
Then they started making deliveries — first in test tubes, then in mice, then in golden retrievers with a genetic mutation similar to the one Conner has. "They were able to run and jump," Jessica says."We saw this with our own eyes. And we just thought, 'Oh gosh, if one day Conner could get a chance to get something like this ...' — it just gave us so much hope."Playing with his brothers, Conner Curran says the gene therapy has really helped him."I can run faster. I stand better," Conner says.
To Conner, the treatment with what he calls"muscle juice" was no big deal."They put a needle in my arm for two hours," he says, when I ask him what it was like.The scientist was with the family in the days after Conner was infused with billions of viruses. He was also there days later when Conner became feverish and stopped eating — a common reaction to this type of treatment.
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