title: [Cell therapy for inherited diseases of the hematopoietic system].
abstract: Cell therapy was born in 1968 with the first allogeneic transplantation of hematopoietic stem cells for two immune deficiency disorders: the Wiskott-Aldrich syndrome and the Severe Combined ImmunoDeficiency (SCID). From this pioneering experience, thousands of patients affected with inherited or acquired diseases of the hematopoietic system have benefited from this therapeutic approach. Unfortunately, immunologic obstacles, represented by the compatibility in the major histocompatibility HLA system, still dictate today important limitations for a larger therapeutic utilization of hematopoietic stem cells (HSC). In this review, we have summarized the difficulties and the scientific advances leading us to improve the clinical results; the therapeutic research's track for primary immunodeficiencies is also discussed.
authors.affiliation: Inserm, U768, Paris & université René-Descartes (Paris-5), hôpital Necker-Enfants-Malades, 149, rue de Sevres, 75743 Paris cedex 15, France. m.cavazzana@nck.aphp.fr
authors.forename: Marina
authors.initials: M
authors.lastname: Cavazzana-Calvo
authors.forename: Liliane
authors.initials: L
authors.lastname: Dal-Cortivo
authors.forename: Isabelle
authors.initials: I
authors.lastname: André-Schmutz
authors.forename: Salima
authors.initials: S
authors.lastname: Hacein-Bey Abina
authors.forename: Alain
authors.initials: A
authors.lastname: Fischer
mesh_terms: D000818:Animals; D006084:Graft Rejection / Q000503:physiopathology; D006680:HLA Antigens / Q000276:immunology / Q000502:physiology; D006402:Hematologic Diseases / Q000235:genetics* / Q000628:therapy*; D006410:Hematopoiesis / Q000502:physiology*; D006413:Hematopoietic System / Q000502:physiology*; D049673:History, 20th Century; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000628:therapy; D033581:Stem Cell Transplantation / Q000266:history
chemical_list: D006680:HLA Antigens