{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T16:21:06Z","timestamp":1790871666559,"version":"4.1.0"},"reference-count":23,"publisher":"MathDoc\/Centre Mersenne","issue":"6-7","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"abstract":"<jats:p>La th\u00e9rapie cellulaire est n\u00e9e avec la premi\u00e8re greffe allog\u00e9nique de cellules souches h\u00e9matopo\u00ef\u00e9tiques r\u00e9alis\u00e9e en 1968 pour deux d\u00e9ficits immunitaires : le syndrome de Wiskott\u2013Aldrich et une forme de d\u00e9ficit immunitaire combin\u00e9 s\u00e9v\u00e8re (SCID). Depuis cette \u00e9tape historique, des milliers de patients atteints de maladies h\u00e9r\u00e9ditaires ou acquises du syst\u00e8me h\u00e9matopo\u00ef\u00e9tique ont b\u00e9n\u00e9fici\u00e9 de cette approche th\u00e9rapeutique. Malheureusement, les obstacles immunologiques repr\u00e9sent\u00e9s par la compatibilit\u00e9 dans le syst\u00e8me majeur d'histocompatibilit\u00e9 (HLA) imposent encore aujourd'hui des limitations importantes \u00e0 une utilisation th\u00e9rapeutique plus large encore des cellules souches h\u00e9matopo\u00ef\u00e9tiques (CSH). Ce texte fait le point sur les difficult\u00e9s rencontr\u00e9es et sur les progr\u00e8s scientifiques, qui laissent esp\u00e9rer une am\u00e9lioration des r\u00e9sultats cliniques ; les pistes de recherche th\u00e9rapeutique concernant les d\u00e9ficits primitifs du syst\u00e8me immunitaire sont \u00e9galement discut\u00e9es.<\/jats:p>","DOI":"10.1016\/j.crvi.2007.04.003","type":"journal-article","created":{"date-parts":[[2007,5,16]],"date-time":"2007-05-16T08:46:03Z","timestamp":1179305163000},"page":"538-542","source":"Crossref","is-referenced-by-count":3,"title":["La th\u00e9rapie cellulaire des maladies h\u00e9r\u00e9ditaires du syst\u00e8me h\u00e9matopo\u00ef\u00e9tique"],"prefix":"10.5802","volume":"330","author":[{"given":"Marina","family":"Cavazzana-Calvo","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Liliane","family":"Dal-Cortivo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Isabelle","family":"Andr\u00e9-Schmutz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Salima","family":"Hacein-Bey Abina","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alain","family":"Fischer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"3842","published-online":{"date-parts":[[2007,5,15]]},"reference":[{"key":"key2025102316293387279_1","first-page":"459","author":"Buckley, R.H.","year":"1999","unstructured":"[1] Buckley, R.H.; Fischer, A. 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Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968\u201399, Lancet, Volume 361 (2003) no. 9357, pp. 553-560","journal-title":"Lancet"},{"issue":"6","key":"key2025102316293387279_4","doi-asserted-by":"crossref","first-page":"740","DOI":"10.1016\/S0022-3476(99)70291-X","article-title":"Influence of severe combined immunodeficiency phenotype on the outcome of HLA non-identical, T-cell-depleted bone marrow transplantation: a retrospective European survey from the European group for bone marrow transplantation and the european society for immunodeficiency","volume":"134","author":"Bertrand, Y.","year":"1999","unstructured":"[4] Bertrand, Y.; Landais, P.; Friedrich, W.; Gerritsen, B.; Morgan, G.; Fasth, A.; Cavazzana-Calvo, M.; Porta, F.; Cant, A.; Espanol, T.; Muller, S.; Veys, P.; Vossen, J.; Haddad, E.; Fischer, A. Influence of severe combined immunodeficiency phenotype on the outcome of HLA non-identical, T-cell-depleted bone marrow transplantation: a retrospective European survey from the European group for bone marrow transplantation and the european society for immunodeficiency, J. Pediatr., Volume 134 (1999) no. 6, pp. 740-748","journal-title":"J. Pediatr."},{"issue":"10","key":"key2025102316293387279_5","first-page":"3646","article-title":"Long-term immune reconstitution and outcome after HLA-nonidentical T-cell-depleted bone marrow transplantation for severe combined immunodeficiency: A European retrospective study of 116 patients","volume":"91","author":"Haddad, E.","year":"1998","unstructured":"[5] Haddad, E.; Landais, P.; Friedrich, W.; Gerritsen, B.; Cavazzana-Calvo, M.; Morgan, G.; Bertrand, Y.; Fasth, A.; Porta, F.; Cant, A.; Espanol, T.; Muller, S.; Veys, P.; Vossen, J.; Fischer, A. Long-term immune reconstitution and outcome after HLA-nonidentical T-cell-depleted bone marrow transplantation for severe combined immunodeficiency: A European retrospective study of 116 patients, Blood, Volume 91 (1998) no. 10, pp. 3646-3653","journal-title":"Blood"},{"issue":"6","key":"key2025102316293387279_6","doi-asserted-by":"crossref","first-page":"844","DOI":"10.1111\/j.1365-2141.2004.05134.x","article-title":"IL-7 effect on immunological reconstitution after HSCT depends on MHC incompatibility","volume":"126","author":"Andr\u00e9-Schmutz, I.","year":"2004","unstructured":"[6] Andr\u00e9-Schmutz, I.; Bonhomme, D.; Yates, F.; Malassis, M.; Selz, F.; Fischer, A.; Cavazzana-Calvo, M. IL-7 effect on immunological reconstitution after HSCT depends on MHC incompatibility, Br. J. Haematol., Volume 126 (2004) no. 6, pp. 844-851","journal-title":"Br. J. Haematol."},{"issue":"2","key":"key2025102316293387279_7","doi-asserted-by":"crossref","first-page":"879","DOI":"10.1182\/blood-2004-03-0960","article-title":"Improved survival after unrelated donor bone marrow transplantation in children with primary immunodeficiency using a reduced-intensity conditioning regimen","volume":"105","author":"Rao, K.","year":"2005","unstructured":"[7] Rao, K.; Amrolia, P.J.; Jones, A.; Cale, C.M.; Naik, P.; King, D.; Davies, G.E.; Gaspar, H.B.; Veys, P.A. 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Infusion of cytotoxic T cells for the prevention and treatment of Epstein\u2013Barr virus-induced lymphoma in allogeneic transplant recipients, Blood, Volume 92 (1998) no. 5, pp. 1549-1555","journal-title":"Blood"},{"issue":"3","key":"key2025102316293387279_11","doi-asserted-by":"crossref","first-page":"379","DOI":"10.1084\/jem.20040613","article-title":"Adoptive transfer of cytomegalovirus-specific CTL to stem cell transplant patients after selection by HLA-peptide tetramers","volume":"202","author":"Cobbold, M.","year":"2005","unstructured":"[11] Cobbold, M.; Khan, N.; Pourgheysari, B.; Tauro, S.; McDonald, D.; Osman, H.; Assenmacher, M.; Billingham, L.; Steward, C.; Crawley, C.; Olavarria, E.; Goldman, J.; Chakraverty, R.; Mahendra, P.; Craddock, C.; Moss, P.A. Adoptive transfer of cytomegalovirus-specific CTL to stem cell transplant patients after selection by HLA-peptide tetramers, J. Exp. Med., Volume 202 (2005) no. 3, pp. 379-386","journal-title":"J. Exp. Med."},{"issue":"2","key":"key2025102316293387279_12","doi-asserted-by":"crossref","first-page":"102","DOI":"10.1080\/14653240510027118","article-title":"Improving immune reconstitution while preventing GvHD in allogeneic stem cell transplantation","volume":"7","author":"Andr\u00e9-Schmutz, I.","year":"2005","unstructured":"[12] Andr\u00e9-Schmutz, I.; Dal Cortivo, L.; Fischer, A.; Cavazzana-Calvo, M. Improving immune reconstitution while preventing GvHD in allogeneic stem cell transplantation, Cytotherapy, Volume 7 (2005) no. 2, pp. 102-108","journal-title":"Cytotherapy"},{"issue":"8","key":"key2025102316293387279_13","doi-asserted-by":"crossref","first-page":"3041","DOI":"10.1182\/blood.V99.8.3041","article-title":"Murine acute graft-versus-host disease can be prevented by depletion of alloreactive T lymphocytes using activation-induced cell death","volume":"99","author":"Hartwig, U.F.","year":"2002","unstructured":"[13] Hartwig, U.F.; Robbers, M.; Wickenhauser, C.; Huber, C. 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Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector\u201d, Lancet, 364 (2004) no. 9452, pp. 2181-2187","journal-title":"Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector\u201d, Lancet"},{"issue":"16","key":"key2025102316293387279_18","doi-asserted-by":"crossref","first-page":"1185","DOI":"10.1056\/NEJMoa012616","article-title":"Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy","volume":"346","author":"Hacein-Bey-Abina, S.","year":"2002","unstructured":"[18] Hacein-Bey-Abina, S.; Le Deist, F.; Carlier, C.; Bouneaud, F.; Hue, C.; de Villartay, J.-P.; Thrasher, A.J.; Wulffraat, N.; Sorensen, R.; Dupuis-Girod, S.; Fischer, A.; Cavazzana-Calvo, M. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy, N. Engl. J. Med., Volume 346 (2002) no. 16, pp. 1185-1193","journal-title":"N. Engl. J. Med."},{"issue":"5644","key":"key2025102316293387279_19","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1126\/science.1088547","article-title":"LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1","volume":"302","author":"Hacein-Bey-Abina, S.","year":"2003","unstructured":"[19] Hacein-Bey-Abina, S.; Von Kalle, C.; Schmidt, M.; McCormack, M.P.; Wulffraat, N.; Leboulch, P.; Lim, A.; Osborne, C.S.; Pawliuk, R.; Morillon, E.; Sorensen, R.; Forster, A.; Fraser, P.; Cohen, J.I.; de Saint Basile, G.; Alexander, I.; Wintergerst, U.; Frebourg, T.; Aurias, A.; Stoppa-Lyonnet, D.; Romana, S.; Radford-Weiss, I.; Gross, F.; Valensi, F.; Delabesse, E.; Macintyre, E.; Sigaux, F.; Soulier, J.; Leiva, L.E.; Wissler, M.; Prinz, C.; Rabbitts, T.H.; Le Deist, F.; Fischer, A.; Cavazzana-Calvo, M. 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