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Gene therapy could correct blood stem cells inside, rather than outside, the body
Nature, Published online: 11 June 2025; doi:10.1038/d41586-025-01779-5Experiments in mice reveal an early postnatal window of opportunity for the effective transfer of genes to blood-cell-producing haematopoietic stem cells by injecting mice with gene-carrying 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.
Nature, Published online: 11 June 2025; doi:10.1038/d41586-025-01779-5Experiments in mice reveal an early postnatal window of opportunity for the effective transfer of genes to blood-cell-producing haematopoietic stem cells by injecting mice with gene-carrying 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.
