title: [Substitution therapy with hematopoietic progenitors in the primary immunodeficiencies].
abstract: Hematopoietic stem-cell transplantation is currently the most appropriate substitution therapy in the most severe forms of primary immunodeficiency diseases (all the variants of SCID, WA, CID etc.). It can achieve total and permanent immunological reconstitution in 60% of patients, depending on histocompatibility, source of the hematopoietic stem cells and the underlying disease. Stem-cell sources may be bone marrow, umbilical cord blood and the peripheral blood of donors previously treated with colony stimulating factors for the mobilization CD34. We discuss the differences in the results obtained in patients treated at the Hospital Materno-Infantil Vall d'Hebron. Gene therapy opens a new era in the treatment of primary immunodeficiency diseases. The first patient to undergo this treatment in the United States of America had adenosine-deaminase deficiency, even though sustained remodeling has not been achieved. The favorable results obtained in patients with SCID by deficit in the gamma chain of the IL-2 receptor in Paris, with more than a year of follow up, suggest that the near future is promising. We also discuss the differences observed according to the vectors used and the underlying disease.
authors: Unidad de Inmunología, Hospital Vall d'Hebron, Barcelona. terespa@hg.vhebron
authors: T
authors: T
authors: Español
mesh_terms: D000243:Adenosine Deaminase / Q000172:deficiency / Q000235:genetics; D016026:Bone Marrow Transplantation; D002675:Child, Preschool; D005312:Fetal Blood / Q000166:cytology; D015316:Genetic Therapy; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000209:etiology; D019650:Hematopoietic Stem Cell Mobilization; D018380:Hematopoietic Stem Cell Transplantation; D006648:Histocompatibility; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000235:genetics / Q000628:therapy*; D007223:Infant; D015375:Receptors, Interleukin-2 / Q000235:genetics; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000628:therapy; D013030:Spain; D014019:Tissue Donors; D016896:Treatment Outcome
chemical_list: D015375:Receptors, Interleukin-2; D000243:Adenosine Deaminase