Lentiviral Gene Therapy in HSCs Restores Lineage-Specific Foxp3 Expression and Suppresses Autoimmunity in a Mouse Model of IPEX Syndrome (Cell Stem Cell)

Masiuk et al. use a lentiviral vector designed from endogenous FOXP3 regulatory elements in HSCs to achieve lineage-specific FoxP3 expression. This strategy restores functional Treg cell development from FoxP3mut HSCs and reverses autoimmunity in vivo. These findings suggest preclinical efficacy for lentiviral gene therapy for the treatment of IPEX syndrome.