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.