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

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 coul...

Descripción completa

Detalles Bibliográficos
Autores: Milani, M, Rio, P
Tipo de recurso: artículo
Fecha de publicación:2025
País:España
Institución:Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT)
Repositorio:Docu-menta. Repositorio Institucional del CIEMAT
Idioma:inglés
OAI Identifier:oai:dnet:documenta___::76bf133fe78af4a6d2d72df8cecd5469
Acceso en línea:https://hdl.handle.net/20.500.14855/5355
Access Level:acceso abierto
Descripción
Sumario: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.