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Gene therapy found effective in hemophilia B

from Reuters

Ten patients with severe hemophilia B have remained cured of the inherited bleeding disorder for as long as three years thanks to gene therapy, according to a new report on the technique in the New England Journal of Medicine. The study updates an earlier one from 2011, in which six volunteers were successfully treated with various doses of the treatment that uses a virus to insert genetic material into the liver. The four additional patients received the highest dose. more


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