Plain Language Summary: PAI-1 deficiency is a rare genetic disorder that causes serious bleeding and heart scarring because the body does not produce enough PAI-1 protein. Researchers developed an experimental mRNA therapy that instructs the liver to make PAI-1. In mouse studies, the treatment successfully increased PAI-1 levels, reduced the breakdown of blood clots, and continued to work with repeated doses. These findings suggest that mRNA therapy could become a preventive treatment for bleeding and cardiac complications in people with PAI-1 deficiency.
