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

Katelyn E. Masiuk, Jennifer Laborada, Maria Grazia Roncarolo, Roger P. Hollis and Donald B. Kohn.

Thursday, January 10, 2019
Published in Cell Stem Cell
Led by Dr. Donald Kohn and Katelyn Masiuk, the researchers engineered a “viral vector” that would turn on the FoxP3 gene only in regulatory T cells — and not in other types of cells.