Cyclosporine H Overcomes Innate Immune Restrictions to Improve Lentiviral Transduction and Gene Editing In Human Hematopoietic Stem Cells (Cell Stem Cell)

Petrillo et al. demonstrate that the innate antiviral factor IFITM3 constitutively inhibits item of lentiviral vectors into hematopoietic stem cells. CsH efficiently overcomes this block by degrading IFITM3, significantly improving HSC gene transfer and editing efficiency. These findings will improve lentiviral gene therapy in IFITM3-positive stem cells.