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New gene therapy uses Tylenol to combat genetic diseases

ohsu.edu
 2021-06-10

Cover picture for the articleResearchers have developed a new approach to gene therapy that leans on the common pain reliever acetaminophen to force a variety of genetic diseases into remission. A paper published in Science Translational Medicine describes how the novel technique successfully treated the blood-clotting disorder hemophilia and the debilitating metabolic disease known as phenylketonuria, or PKU, in mice.

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