Gene therapy could correct blood stem cells inside, rather than outside, the body

Abstract

Experiments in mice reveal an early postnatal window of opportunity for the effective transfer of genes to blood-cellproducing haematopoietic stem cells by injecting mice with genecarrying lentiviral vectors. This approach showed therapeutic benefit in three mouse models of severe diseases, and could expand the applicability of haematopoietic stem-cell gene therapy in the clinic.