[Cell therapy for inherited diseases of the hematopoietic system].
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.
Cavazzana-Calvo
Marina
M
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
Dal-Cortivo
Liliane
L
André-Schmutz
Isabelle
I
Hacein-Bey Abina
Salima
S
Fischer
Alain
A
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
D006680:HLA Antigens