New gene therapy uses Tylenol to combat genetic diseases

New gene therapy uses Tylenol to combat genetic diseases

Researchers 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. The approach uses a benign lentivirus to both correct disease-causing mutations and to insert a new gene that makes liver cells immune to the potentially toxic effects of acetaminophen. The latter enables treated cells to survive -- and kills off untreated cells -- when they’re repeatedly exposed to the over-the-counter medication that’s also known by the brand name Tylenol. This allows the treated cells to multiply and creates a greater portion of liver cells being corrected. “This gene therapy gets rid of liver cells that don’t have a therapeutic gene inserted,” said the paper’s corresponding author, Markus Grompe, M.D., a professor of pediatrics, and molecular and medical genetics in the Oregon Health & Science University School of Medicine. “We’re essentially poisoning untreated cells with …
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