{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,21]],"date-time":"2025-10-21T14:45:38Z","timestamp":1761057938471},"reference-count":85,"publisher":"Elsevier BV","issue":"4","license":[{"start":{"date-parts":[[2004,4,1]],"date-time":"2004-04-01T00:00:00Z","timestamp":1080777600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Journal of Allergy and Clinical Immunology"],"published-print":{"date-parts":[[2004,4]]},"DOI":"10.1016\/j.jaci.2004.01.765","type":"journal-article","created":{"date-parts":[[2004,4,26]],"date-time":"2004-04-26T09:21:14Z","timestamp":1082971274000},"page":"595-603","source":"Crossref","is-referenced-by-count":27,"title":["Successes and risks of gene therapy in primary immunodeficiencies"],"prefix":"10.1016","volume":"113","author":[{"given":"Javier","family":"Chinen","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jennifer M","family":"Puck","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.jaci.2004.01.765_BIB1","doi-asserted-by":"crossref","first-page":"206","DOI":"10.1034\/j.1600-065X.2002.18517.x","article-title":"Primary immunodeficiency disease: dissectors of the immune system","volume":"185","author":"Buckley","year":"2002","journal-title":"Immunol Rev"},{"key":"10.1016\/j.jaci.2004.01.765_BIB2","series-title":"Primary immunodeficiency diseases, a molecular and genetic approach","first-page":"3","article-title":"Genetically determined immunodeficiency diseases","author":"Smith","year":"1999"},{"key":"10.1016\/j.jaci.2004.01.765_BIB3","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1046\/j.1365-2249.2003.02110.x","article-title":"Primary Immunodeficiency diseases: an update","volume":"132","author":"Chapel","year":"2003","journal-title":"Clin Exp Immunol"},{"key":"10.1016\/j.jaci.2004.01.765_BIB4","series-title":"Hematology 2003 (Am Soc Hematol Educ Program Book)","first-page":"314","article-title":"Immunodeficiency disorders","author":"Cooper","year":"2003"},{"key":"10.1016\/j.jaci.2004.01.765_BIB5","doi-asserted-by":"crossref","first-page":"378","DOI":"10.1016\/S0022-3476(97)70199-9","article-title":"Human severe combined immunodeficiency: genetic, phenotypics and functional diversity in one hundred and eight infants","volume":"130","author":"Buckley","year":"1997","journal-title":"J Pediatr"},{"key":"10.1016\/j.jaci.2004.01.765_BIB6","doi-asserted-by":"crossref","first-page":"1821","DOI":"10.1056\/NEJMoa031178","article-title":"Effects of CD3\u03b4 deficiency in maturation of \u03b1\/\u03b2 and \u03b3\/\u03b4 T-cell lineages in severe combined immunodeficiency","volume":"349","author":"Dadi","year":"2003","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jaci.2004.01.765_BIB7","doi-asserted-by":"crossref","first-page":"195","DOI":"10.1016\/S0092-8674(01)00205-7","article-title":"Mutations in the RNA component of RNase MRP cause a pleiotropic human disease, cartilage-hair hypoplasia","volume":"104","author":"Ridanpaa","year":"2001","journal-title":"Cell"},{"key":"10.1016\/j.jaci.2004.01.765_BIB8","doi-asserted-by":"crossref","first-page":"473","DOI":"10.1038\/18997","article-title":"Exposing the human nude phenotype","volume":"398","author":"Frank","year":"1999","journal-title":"Nature"},{"key":"10.1016\/j.jaci.2004.01.765_BIB9","series-title":"Primary immunodeficiency diseases, a molecular and genetic approach","first-page":"448","article-title":"Conventional therapy of primary immunodeficiency diseases","author":"Stiehm","year":"1999"},{"key":"10.1016\/j.jaci.2004.01.765_BIB10","doi-asserted-by":"crossref","first-page":"1366","DOI":"10.1016\/S0140-6736(68)92673-1","article-title":"Immunologic reconstitution of sex-linked lymphopenic immunological deficiency","volume":"2","author":"Gatti","year":"1968","journal-title":"Lancet"},{"key":"10.1016\/j.jaci.2004.01.765_BIB11","doi-asserted-by":"crossref","first-page":"341","DOI":"10.1182\/blood.V61.2.341.341","article-title":"Transplantation of severe combined immunodeficiency with HLA-A, B, D DR incompatible parental marrow cells fractionated by soybean agglutinin and sheep red blood cells","volume":"61","author":"Reisner","year":"1983","journal-title":"Blood"},{"key":"10.1016\/j.jaci.2004.01.765_BIB12","doi-asserted-by":"crossref","first-page":"508","DOI":"10.1056\/NEJM199902183400703","article-title":"Hematopoietic stem cell transplantation for the treatment of severe combined immunodeficiency","volume":"340","author":"Buckley","year":"1999","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jaci.2004.01.765_BIB13","doi-asserted-by":"crossref","first-page":"553","DOI":"10.1016\/S0140-6736(03)12513-5","article-title":"Long-term survival and transplantation of hematopoietic stem cells for immunodeficiencies: report of the European experience 1968-1999","volume":"361","author":"Antoine","year":"2003","journal-title":"Lancet"},{"key":"10.1016\/j.jaci.2004.01.765_BIB14","first-page":"111","article-title":"T cell depleted haploidentical bone marrow transplantation for the treatment of children with severe combined immunodeficiency","volume":"48","author":"Smogorzewska","year":"2000","journal-title":"Arch Immunol Ther Exp (Warsz)"},{"key":"10.1016\/j.jaci.2004.01.765_BIB15","doi-asserted-by":"crossref","first-page":"872","DOI":"10.1182\/blood.V99.3.872","article-title":"Hematopoietic stem cell transplantation for SCID in the neonatal period leads to superior thymic output and improved survival","volume":"99","author":"Myers","year":"2002","journal-title":"Blood"},{"key":"10.1016\/j.jaci.2004.01.765_BIB16","doi-asserted-by":"crossref","first-page":"886","DOI":"10.1046\/j.1365-2141.2003.04390.x","article-title":"Risks of mortality in children admitted to the paediatric intensive care unit after haematopoietic stem cell transplantation","volume":"121","author":"Tomaske","year":"2003","journal-title":"Br J Haematol"},{"key":"10.1016\/j.jaci.2004.01.765_BIB17","series-title":"Primary immunodeficiency diseases, a molecular and genetic approach","first-page":"459","article-title":"Bone marrow transplantation for primary immunodeficiency diseases","author":"Buckley","year":"1999"},{"key":"10.1016\/j.jaci.2004.01.765_BIB18","doi-asserted-by":"crossref","first-page":"1325","DOI":"10.1056\/NEJM200005043421804","article-title":"Thymic function after hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency","volume":"342","author":"Patel","year":"2000","journal-title":"N Engl J Med"},{"issue":"Part 1","key":"10.1016\/j.jaci.2004.01.765_BIB19","first-page":"1800","article-title":"Absence of B-cell, NK-cell myeloid or CD34 cell engraftment in some X-linked severe combined immunodeficiency (XSCID) patients following haploidentical transplant","volume":"100","author":"Hay","year":"2002","journal-title":"Blood"},{"key":"10.1016\/j.jaci.2004.01.765_BIB20","doi-asserted-by":"crossref","first-page":"1104","DOI":"10.1182\/blood.V88.3.1104.1104","article-title":"Granulocyte colony-stimulating factor recruitment of CD34+ progenitors to peripheral blood: impaired mobilization in chronic granulomatous disease and adenosine deaminase\u2013deficient severe combined immunodeficiency disease patients","volume":"88","author":"Sekhsaria","year":"1996","journal-title":"Blood"},{"key":"10.1016\/j.jaci.2004.01.765_BIB21","doi-asserted-by":"crossref","first-page":"2728","DOI":"10.1182\/blood-2003-02-0663","article-title":"Mobilization of hematopoietic progenitor cells in healthy volunteers by AMD3100, a CXCR4 antagonist","volume":"102","author":"Liles","year":"2003","journal-title":"Blood"},{"key":"10.1016\/j.jaci.2004.01.765_BIB22","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/S0167-4889(03)00024-7","article-title":"Current developments in the design of onco-retrovirus and lentivirus vector systems for hematopoietic cell gene therapy","volume":"1640","author":"Brenner","year":"2003","journal-title":"Biochim Biophys Acta"},{"key":"10.1016\/j.jaci.2004.01.765_BIB23","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1034\/j.1600-065X.2000.17802.x","article-title":"Hematopoietic stem cell gene therapy: towards clinically significant gene transfer efficiency","volume":"178","author":"Heim","year":"2000","journal-title":"Immunol Rev"},{"key":"10.1016\/j.jaci.2004.01.765_BIB24","doi-asserted-by":"crossref","first-page":"669","DOI":"10.1126\/science.288.5466.669","article-title":"Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease","volume":"288","author":"Cavazzana-Calvo","year":"2000","journal-title":"Science"},{"key":"10.1016\/j.jaci.2004.01.765_BIB25","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","year":"2003","journal-title":"Science"},{"key":"10.1016\/j.jaci.2004.01.765_BIB26","series-title":"Primary immunodeficiency diseases, a molecular and genetic approach","first-page":"121","article-title":"Immunodeficiency disease due to deficiency of adenosine deaminase","author":"Hirschhorn","year":"1999"},{"key":"10.1016\/j.jaci.2004.01.765_BIB27","doi-asserted-by":"crossref","first-page":"663","DOI":"10.1073\/pnas.83.17.6563","article-title":"Correction of adenosine deaminase deficiency in human T and B cells using retroviral gene transfer","volume":"83","author":"Kantoff","year":"1986","journal-title":"Proc Natl Acad Sci U S A"},{"key":"10.1016\/j.jaci.2004.01.765_BIB28","doi-asserted-by":"crossref","first-page":"385","DOI":"10.1038\/322385a0","article-title":"Expression of human adenosine deaminase in murine haematopoietic progenitor cells following retroviral transfer","volume":"322","author":"Belmont","year":"1986","journal-title":"Nature"},{"key":"10.1016\/j.jaci.2004.01.765_BIB29","doi-asserted-by":"crossref","first-page":"475","DOI":"10.1126\/science.270.5235.475","article-title":"T-lymphocyte directed gene therapy for ADA-SCD: initial trial results after 4 years","volume":"270","author":"Blaese","year":"1995","journal-title":"Science"},{"key":"10.1016\/j.jaci.2004.01.765_BIB30","doi-asserted-by":"crossref","first-page":"2563","DOI":"10.1182\/blood-2002-09-2800","article-title":"Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial","volume":"101","author":"Muul","year":"2003","journal-title":"Blood"},{"key":"10.1016\/j.jaci.2004.01.765_BIB31","doi-asserted-by":"crossref","first-page":"589","DOI":"10.1056\/NEJM198703053161005","article-title":"Treatment of adenosine deaminase deficiency with polyethylene glycol-modified adenosine deaminase","volume":"316","author":"Hershfield","year":"1987","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jaci.2004.01.765_BIB32","doi-asserted-by":"crossref","first-page":"1605","DOI":"10.1089\/10430340260201699","article-title":"Immune response to fetal calf serum by two adenosine deaminase-deficient patients after T cell gene therapy","volume":"13","author":"Tuschong","year":"2002","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.jaci.2004.01.765_BIB33","doi-asserted-by":"crossref","first-page":"470","DOI":"10.1126\/science.270.5235.470","article-title":"gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients","volume":"270","author":"Bordignon","year":"1995","journal-title":"Science"},{"key":"10.1016\/j.jaci.2004.01.765_BIB34","doi-asserted-by":"crossref","first-page":"1017","DOI":"10.1038\/nm1095-1017","article-title":"Engraftment of gene modified cells from umbilical cord blood in neonates with adenosine deaminase deficiency","volume":"1","author":"Kohn","year":"1995","journal-title":"Nat Med"},{"key":"10.1016\/j.jaci.2004.01.765_BIB35","first-page":"179","article-title":"Bone marrow gene transfer in three patients with adenosine deaminase deficiency","volume":"3","author":"Hoogerbrugge","year":"1996","journal-title":"Gene Ther"},{"key":"10.1016\/j.jaci.2004.01.765_BIB36","doi-asserted-by":"crossref","first-page":"775","DOI":"10.1038\/nm0798-775","article-title":"T-lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA deficient neonates","volume":"4","author":"Kohn","year":"1998","journal-title":"Nat Med"},{"key":"10.1016\/j.jaci.2004.01.765_BIB37","doi-asserted-by":"crossref","first-page":"2410","DOI":"10.1126\/science.1070104","article-title":"Correction of ADA-SCID by stem cell gene therapy combined with non-myeloablative conditioning","volume":"296","author":"Aiuti","year":"2002","journal-title":"Science"},{"issue":"part I","key":"10.1016\/j.jaci.2004.01.765_BIB38","first-page":"154a","article-title":"Safety and efficacy of stem cell gene therapy combined with nonmyeloablative conditioning for the treatment of ADA-SCID","volume":"102","author":"Aiuti","year":"2003","journal-title":"Blood"},{"key":"10.1016\/j.jaci.2004.01.765_BIB39","doi-asserted-by":"crossref","first-page":"147","DOI":"10.1016\/0092-8674(93)90167-O","article-title":"Interleukin-2 receptor \u03b3-chain mutation results in X-linked severe combined immunodeficiency in humans","volume":"73","author":"Noguchi","year":"1993","journal-title":"Cell"},{"key":"10.1016\/j.jaci.2004.01.765_BIB40","doi-asserted-by":"crossref","first-page":"1099","DOI":"10.1093\/hmg\/2.8.1099","article-title":"The interleukin-2 receptor gamma chain maps to Xq13.1 and is mutated in X-linked severe combined immunodeficiency, XSCID1","volume":"2","author":"Puck","year":"1992","journal-title":"Hum Mol Genet"},{"key":"10.1016\/j.jaci.2004.01.765_BIB41","series-title":"Primary immunodeficiency diseases, a molecular and genetic approach","first-page":"99","article-title":"X-linked severe combined immunodeficiency","author":"Puck","year":"1999"},{"key":"10.1016\/j.jaci.2004.01.765_BIB42","doi-asserted-by":"crossref","first-page":"1395","DOI":"10.1172\/JCI112967","article-title":"Carrier detection in X-linked severe combined immunodeficiency based on patterns of X chromosome inactivation","volume":"79","author":"Puck","year":"1987","journal-title":"J Clin Invest"},{"key":"10.1016\/j.jaci.2004.01.765_BIB43","doi-asserted-by":"crossref","first-page":"1563","DOI":"10.1056\/NEJM199611213352104","article-title":"Atypical X-linked severe combined immunodeficiency due to possible spontaneous reversion of the genetic defect in T cells","volume":"335","author":"Stephan","year":"1996","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jaci.2004.01.765_BIB44","doi-asserted-by":"crossref","first-page":"3108","DOI":"10.1182\/blood.V87.8.3108.bloodjournal8783108","article-title":"Gamma c gene transfer into SCID X1 patients' B-cell lines restores normal high affinity interleukin-2 receptor expression and function","volume":"87","author":"Hacein-Bey","year":"1996","journal-title":"Blood"},{"key":"10.1016\/j.jaci.2004.01.765_BIB45","doi-asserted-by":"crossref","first-page":"3103","DOI":"10.1182\/blood.V87.8.3103.bloodjournal8783103","article-title":"Correction of interleukin-2 receptor function in X-SCID lymphoblastoid cells by retrovirally mediated transfer of the gamma-c gene","volume":"87","author":"Taylor","year":"1996","journal-title":"Blood"},{"key":"10.1016\/j.jaci.2004.01.765_BIB46","doi-asserted-by":"crossref","first-page":"3097","DOI":"10.1182\/blood.V87.8.3097.bloodjournal8783097","article-title":"Retroviral-mediated gene correction for X-linked severe combined immunodeficiency (SCID)","volume":"87","author":"Candotti","year":"1996","journal-title":"Blood"},{"key":"10.1016\/j.jaci.2004.01.765_BIB47","doi-asserted-by":"crossref","first-page":"145","DOI":"10.1006\/mthe.1999.0020","article-title":"Lymphoid development and function in X-linked severe combined immunodeficiency mice after stem cell gene therapy","volume":"1","author":"Otsu","year":"2000","journal-title":"Mol Ther"},{"key":"10.1016\/j.jaci.2004.01.765_BIB48","doi-asserted-by":"crossref","first-page":"3071","DOI":"10.1182\/blood.V95.10.3071","article-title":"Stable and functional lymphoid reconstitution of common cytokine receptor gamma chain deficient mice by retroviral-mediated gene transfer","volume":"95","author":"Soudais","year":"2000","journal-title":"Blood"},{"key":"10.1016\/j.jaci.2004.01.765_BIB49","doi-asserted-by":"crossref","first-page":"72","DOI":"10.1182\/blood.V100.1.72","article-title":"Retroviral transduction of ILRG into CD34 from XSCID patients permits human T and B cell development in sheep chimeras","volume":"100","author":"Tsai","year":"2002","journal-title":"Blood"},{"key":"10.1016\/j.jaci.2004.01.765_BIB50","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","year":"2002","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jaci.2004.01.765_BIB51","first-page":"1999","article-title":"Immune recovery following retroviral mediated common gamma chain gene therapy for X-linked severe combined immunodeficiency","volume":"10","author":"Thrasher","year":"2003","journal-title":"Gene Ther"},{"key":"10.1016\/j.jaci.2004.01.765_BIB52","doi-asserted-by":"crossref","first-page":"255","DOI":"10.1056\/NEJM200301163480314","article-title":"A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency","volume":"348","author":"Hacein-Bey-Abina","year":"2003","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jaci.2004.01.765_BIB53","doi-asserted-by":"crossref","first-page":"170","DOI":"10.1097\/00005792-200005000-00004","article-title":"Genetic, biochemical, and clinical features of chronic granulomatous disease","volume":"79","author":"Segal","year":"2000","journal-title":"Medicine"},{"key":"10.1016\/j.jaci.2004.01.765_BIB54","doi-asserted-by":"crossref","first-page":"881","DOI":"10.1056\/NEJM200103223441203","article-title":"Treatment of chronic granulomatous disease with nonmyeloablative conditioning and a T-cell-depleted hematopoietic allograft","volume":"344","author":"Horwitz","year":"2001","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jaci.2004.01.765_BIB55","doi-asserted-by":"crossref","first-page":"2268","DOI":"10.1182\/blood.V89.7.2268","article-title":"Enhanced host defense after gene transfer in the murine p47phox-deficient model of chronic granulomatous disease","volume":"89","author":"Mardiney","year":"1997","journal-title":"Blood"},{"key":"10.1016\/j.jaci.2004.01.765_BIB56","doi-asserted-by":"crossref","first-page":"914","DOI":"10.1182\/blood.V94.3.914.415a11_914_922","article-title":"Long term correction of phagocyte NADPH oxidase activity by retroviral-mediated gene transfer in murine X-linked chronic granulomatous disease","volume":"94","author":"Dinauer","year":"1999","journal-title":"Blood"},{"key":"10.1016\/j.jaci.2004.01.765_BIB57","doi-asserted-by":"crossref","first-page":"1663","DOI":"10.1182\/blood.V87.5.1663.1663","article-title":"Mutations in the X-linked and autosomal recessive forms of chronic granulomatous disease","volume":"87","author":"Roos","year":"1996","journal-title":"Blood"},{"key":"10.1016\/j.jaci.2004.01.765_BIB58","doi-asserted-by":"crossref","first-page":"12133","DOI":"10.1073\/pnas.94.22.12133","article-title":"Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease","volume":"94","author":"Malech","year":"1997","journal-title":"Proc Natl Acad Sci U S A"},{"key":"10.1016\/j.jaci.2004.01.765_BIB59","series-title":"Primary immunodeficiency diseases, a molecular and genetic approach","first-page":"375","article-title":"Cell adhesion and leukocyte adhesion defects","author":"Etzioni","year":"1999"},{"key":"10.1016\/j.jaci.2004.01.765_BIB60","first-page":"383","article-title":"Gene therapy for leukocyte adhesion deficiency","volume":"2","author":"Bauer","year":"2000","journal-title":"Curr Opin Mol Ther"},{"key":"10.1016\/j.jaci.2004.01.765_BIB61","doi-asserted-by":"crossref","first-page":"585","DOI":"10.1016\/S0952-7915(03)00112-2","article-title":"Wiskott-Aldrich Syndrome: a model for defective actin reorganization, cell trafficking and synapse formation","volume":"15","author":"Notarangelo","year":"2003","journal-title":"Curr Opin Immunol"},{"key":"10.1016\/j.jaci.2004.01.765_BIB62","doi-asserted-by":"crossref","first-page":"1598","DOI":"10.1182\/blood.V97.6.1598","article-title":"Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program","volume":"97","author":"Filipovich","year":"2001","journal-title":"Blood"},{"key":"10.1016\/j.jaci.2004.01.765_BIB63","doi-asserted-by":"crossref","first-page":"2369","DOI":"10.1182\/blood.V75.12.2369.2369","article-title":"Atypical presentation of Wiskott-Aldrich syndrome: diagnosis in two unrelated males based on studies of maternal T cell X chromosome inactivation","volume":"75","author":"Puck","year":"1990","journal-title":"Blood"},{"key":"10.1016\/j.jaci.2004.01.765_BIB64","doi-asserted-by":"crossref","first-page":"8697","DOI":"10.1073\/pnas.151260498","article-title":"Somatic mosaicism in WAS suggests in vivo reversion by a DNA slippage mechanism","volume":"98","author":"Wada","year":"2001","journal-title":"Proc Natl Acad Sci U S A"},{"key":"10.1016\/j.jaci.2004.01.765_BIB65","doi-asserted-by":"crossref","first-page":"1039","DOI":"10.1089\/104303402753812449","article-title":"Retrovirus-mediated WASP gene transfer corrects Wiskott-Aldrich syndrome T-cell dysfunction","volume":"13","author":"Wada","year":"2002","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.jaci.2004.01.765_BIB66","doi-asserted-by":"crossref","first-page":"803","DOI":"10.1038\/sj.gt.3301950","article-title":"Functional correction of T-cells derived from patients with the Wiskott-Aldrich syndrome (WAS) by transduction with an oncoretroviral vector encoding the WAS protein","volume":"10","author":"Strom","year":"2003","journal-title":"Gene Ther"},{"key":"10.1016\/j.jaci.2004.01.765_BIB67","doi-asserted-by":"crossref","first-page":"1170","DOI":"10.1038\/sj.gt.3300926","article-title":"Retrovirus-mediated WASP gene transfer corrects defective actin polymerization in B cell lines from Wiskott-Aldrich syndrome carrying \u2018null\u2019 mutations","volume":"6","author":"Candotti","year":"1999","journal-title":"Gene Ther"},{"key":"10.1016\/j.jaci.2004.01.765_BIB68","doi-asserted-by":"crossref","first-page":"81","DOI":"10.1016\/S1074-7613(00)80590-7","article-title":"Wiskott-Aldrich syndrome protein deficient mice reveal a role for WASP in T but not T cell activation","volume":"9","author":"Snapper","year":"1998","journal-title":"Immunity"},{"key":"10.1016\/j.jaci.2004.01.765_BIB69","doi-asserted-by":"crossref","first-page":"589","DOI":"10.1016\/S0022-3476(99)70246-5","article-title":"Early and prolonged intra venous immunoglobulin replacement therapy in childhood agammaglobulinemia: a retrospective survey of 31 patients","volume":"134","author":"Quartier","year":"1999","journal-title":"J Pediatr"},{"key":"10.1016\/j.jaci.2004.01.765_BIB70","doi-asserted-by":"crossref","first-page":"90","DOI":"10.1016\/S1521-6616(03)00044-5","article-title":"Reconstitution of B cell function in murine models of immunodeficiency","volume":"107","author":"Porpiglia","year":"2003","journal-title":"Clin Immunol"},{"key":"10.1016\/j.jaci.2004.01.765_BIB71","doi-asserted-by":"crossref","first-page":"3358","DOI":"10.1182\/blood.V94.10.3358.422k04_3358_3365","article-title":"Correction of X-linked immunodeficient mice by competitive reconstitution with limiting numbers of normal bone marrow cells","volume":"94","author":"Rohrer","year":"1999","journal-title":"Blood"},{"key":"10.1016\/j.jaci.2004.01.765_BIB72","doi-asserted-by":"crossref","first-page":"1299","DOI":"10.1038\/sj.cgt.0234","article-title":"Lymphoid abnormalities in CD40 ligand transgenic mice suggest the need for tight regulation in gene therapy approaches to hyper immunoglobulin M (IgM) syndrome","volume":"7","author":"Sacco","year":"2000","journal-title":"Cancer Gene Ther"},{"key":"10.1016\/j.jaci.2004.01.765_BIB73","doi-asserted-by":"crossref","first-page":"1253","DOI":"10.1038\/3233","article-title":"Thymic lymphoproliferative disease after successful correction of CD40 ligand deficiency by gene transfer in mice","volume":"4","author":"Brown","year":"1998","journal-title":"Nat Med"},{"key":"10.1016\/j.jaci.2004.01.765_BIB74","doi-asserted-by":"crossref","first-page":"65","DOI":"10.1038\/377065a0","article-title":"Mutations of Jak-3 gene in patients with autosomal severe combined immunodeficiency (SCID)","volume":"377","author":"Macchi","year":"1995","journal-title":"Nature"},{"key":"10.1016\/j.jaci.2004.01.765_BIB75","doi-asserted-by":"crossref","first-page":"2353","DOI":"10.1089\/104303400750038462","article-title":"Self-selection by genetically modified committed lymphocyte precursors reverses the phenotype of Jak-3 deficient mice without myeloablation","volume":"11","author":"Bunting","year":"2000","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.jaci.2004.01.765_BIB76","doi-asserted-by":"crossref","first-page":"3942","DOI":"10.1182\/blood-2002-03-0782","article-title":"Gene therapy of RAG-2-\/- mice: sustained correction of the immunodeficiency","volume":"100","author":"Yates","year":"2002","journal-title":"Blood"},{"key":"10.1016\/j.jaci.2004.01.765_BIB77","first-page":"1794s","article-title":"The effect of chromosomal translocations in acute leukemias: the LMO2 paradigm in transcription and development","volume":"59","author":"Rabbitts","year":"1999","journal-title":"Cancer Res"},{"key":"10.1016\/j.jaci.2004.01.765_BIB78","doi-asserted-by":"crossref","first-page":"180","DOI":"10.1016\/S1525-0016(03)00212-0","article-title":"American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells","volume":"8","author":"Kohn","year":"2003","journal-title":"Mol Ther"},{"key":"10.1016\/j.jaci.2004.01.765_BIB79","doi-asserted-by":"crossref","first-page":"346","DOI":"10.1038\/nrg1066","article-title":"Progress and problems with the use of viral vectors for gene therapy","volume":"4","author":"Thomas","year":"2003","journal-title":"Nat Rev Genet"},{"key":"10.1016\/j.jaci.2004.01.765_BIB80","doi-asserted-by":"crossref","first-page":"860","DOI":"10.1038\/35057062","article-title":"Initial sequencing and analysis of the human genome","volume":"409","author":"International Human Genome Sequencing Consortium","year":"2001","journal-title":"Nature"},{"key":"10.1016\/j.jaci.2004.01.765_BIB81","doi-asserted-by":"crossref","first-page":"521","DOI":"10.1016\/S0092-8674(02)00864-4","article-title":"HIV-1 integration in the human genome favors active genes and local hotspots","volume":"110","author":"Schroder","year":"2002","journal-title":"Cell"},{"key":"10.1016\/j.jaci.2004.01.765_BIB82","doi-asserted-by":"crossref","first-page":"2191","DOI":"10.1182\/blood-2002-02-0627","article-title":"Retroviral integration occurs into preferred genomic targets of human bone marrow repopulating cells","volume":"101","author":"Laufs","year":"2002","journal-title":"Blood"},{"key":"10.1016\/j.jaci.2004.01.765_BIB83","doi-asserted-by":"crossref","first-page":"1749","DOI":"10.1126\/science.1083413","article-title":"Transcription start regions in the human genome are favored targets for MLV integration","volume":"300","author":"Wu","year":"2003","journal-title":"Science"},{"key":"10.1016\/j.jaci.2004.01.765_BIB84","doi-asserted-by":"crossref","first-page":"9150","DOI":"10.1073\/pnas.160159597","article-title":"A chromatin insulator protects retrovirus vectors from chromosomal position effects","volume":"97","author":"Emery","year":"2000","journal-title":"Proc Natl Acad Sci U S A"},{"key":"10.1016\/j.jaci.2004.01.765_BIB85","doi-asserted-by":"crossref","first-page":"4679","DOI":"10.1128\/JVI.74.10.4679-4687.2000","article-title":"The cHS4 insulator increases the probability of retroviral expression at random chromosomal integration","volume":"74","author":"Rivella","year":"2000","journal-title":"J Virol"}],"container-title":["Journal of Allergy and Clinical Immunology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0091674904009212?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0091674904009212?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2020,4,1]],"date-time":"2020-04-01T19:14:16Z","timestamp":1585768456000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0091674904009212"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2004,4]]},"references-count":85,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2004,4]]}},"alternative-id":["S0091674904009212"],"URL":"https:\/\/doi.org\/10.1016\/j.jaci.2004.01.765","relation":{},"ISSN":["0091-6749"],"issn-type":[{"value":"0091-6749","type":"print"}],"subject":[],"published":{"date-parts":[[2004,4]]}}}