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Gene therapy is showing promise for treating one of the most common genetic disorders.
Results of a study published Wednesday show that 15 of 22 patients with beta-thalassemia who got gene therapy were able to stop or sharply reduce the regular blood transfusions they had needed to alleviate their life-threatening anemia. There were no serious side effects.
"We're extraordinarily excited about these early results," says Alexis Thompson, a professor of pediatrics at the Northwestern University's Feinberg School of Medicine, who helped with the study released Wednesday.
"For the first time ever, we have a treatment that we might offer to all our patients," says Mark Walters of the University of California, San Francisco, who also helped conduct the study.
The researchers stress, however, that more research is needed to fully evaluate how well the treatment works and how safe it is.
Still, the company that's developing the treatment, Bluebird Bio of Cambridge, Mass., plans to seek approval of the treatment in Europe by the end of the year, a spokeswoman said in an email.
An estimated 288,000 people...