Researchers have ameliorated both monogenic and complex inflammation-driven diseases through transplantation of hematopoietic stem cells with an inserted IL-1 receptor antagonist (IL-1RA) gene. The team showed that in animal models the transplanted cells worked better than monoclonal antibodies to reduce symptoms in systemic autoinflammatory diseases (SAIDs), a group of childhood-onset, lifelong diseases that vary in severity depending on the underlying mutation, but can be life-threatening.
A Sino-U.S. study has shown that the increased expression of the small secreted glycoprotein follistatin-like 1 is critical in the pathogenesis of multiple fibrotic and systemic autoimmune diseases, the authors reported in the January 6, 2021, print edition of MolecularTherapy after earlier publication online.