title: Innovations Needed for Effective Implementation ofEx VivoGene Therapies.
authors.affiliation: Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; INSERM, Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hôpitaux de Paris, Paris, France; Imagine Institute, Paris Descartes-Sorbonne Paris Cité University, Paris, Italy; INSERM UMR 1163, Laboratory of Human Lymphohematopoiesis, Paris, France.
authors.forename: Marina
authors.initials: M
authors.lastname: Cavazzana
keywords: Wiskott–Aldrich syndrome; dyserythropoiesis; gene therapy; genome-editing technology; hematopoietic stem progenitor cells; hemoglobinopathies