IPEX syndrome is a severe autoimmune disease with limited treatment options caused by mutations in the forkhead box protein 3 (FOXP3) gene, which plays a critical role in immune regulation.
Despite their rapidly expanding therapeutic potential, human pluripotent stem cell (hPSC)-derived cell therapies continue to have serious safety risks.
Researchers at Stanford have developed methods to overcome the limited packaging capacity of adeno-associated virus (AAV) vectors and enable their use in integration of large transgenes.