New gene therapy uses Tylenol to combat genetic diseases

Novel approach corrects disease-causing mutations by making treated livers cells immune to acetaminophen

By Franny White June 10, 2021 Portland, Oregon

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Gene therapy uses pain reliever to combat genetic diseases

This infographic illustrates how Oregon Health & Science University’s new gene therapy method both corrects genetic mutations and inserts 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 image was created with BioRender.com. (OHSU)

Markus Grompe, M.D.

Mark Grompe, M.D., and colleagues at Oregon Health & Science University have developed a new approach to gene therapy that uses the common pain reliever acetaminophen to force a variety of genetic diseases into remission. (OHSU)