{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,24]],"date-time":"2026-09-24T16:15:50Z","timestamp":1790266550434,"version":"4.1.0"},"reference-count":38,"publisher":"Elsevier BV","issue":"2","license":[{"start":{"date-parts":[[2013,8,1]],"date-time":"2013-08-01T00:00:00Z","timestamp":1375315200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Gene"],"published-print":{"date-parts":[[2013,8]]},"DOI":"10.1016\/j.gene.2013.03.092","type":"journal-article","created":{"date-parts":[[2013,4,10]],"date-time":"2013-04-10T11:19:19Z","timestamp":1365592759000},"page":"170-173","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":46,"title":["Gene therapy of primary T cell immunodeficiencies"],"prefix":"10.1016","volume":"525","author":[{"given":"Alain","family":"Fischer","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Salima","family":"Hacein-Bey-Abina","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marina","family":"Cavazzana-Calvo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.gene.2013.03.092_bb0010","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 nonmyeloablative conditioning","volume":"296","author":"Aiuti","year":"2002","journal-title":"Science"},{"key":"10.1016\/j.gene.2013.03.092_bb0005","doi-asserted-by":"crossref","first-page":"447","DOI":"10.1056\/NEJMoa0805817","article-title":"Gene therapy for immunodeficiency due to adenosine deaminase deficiency","volume":"360","author":"Aiuti","year":"2009","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.gene.2013.03.092_bb0015","doi-asserted-by":"crossref","first-page":"1490","DOI":"10.1038\/mt.2008.118","article-title":"Stable and functional lymphoid reconstitution in artemis-deficient mice following lentiviral artemis gene transfer into hematopoietic stem cells","volume":"16","author":"Benjelloun","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.gene.2013.03.092_bb0020","doi-asserted-by":"crossref","first-page":"1345","DOI":"10.1126\/science.1191536","article-title":"Aryl hydrocarbon receptor antagonists promote the expansion of human hematopoietic stem cells","volume":"329","author":"Boitano","year":"2010","journal-title":"Science"},{"key":"10.1016\/j.gene.2013.03.092_bb0025","doi-asserted-by":"crossref","first-page":"1918","DOI":"10.1056\/NEJMoa1003548","article-title":"Stem-cell gene therapy for the Wiskott\u2013Aldrich syndrome","volume":"363","author":"Boztug","year":"2010","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.gene.2013.03.092_bb0030","doi-asserted-by":"crossref","first-page":"3635","DOI":"10.1182\/blood-2012-02-400937","article-title":"Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans","volume":"120","author":"Candotti","year":"2012","journal-title":"Blood"},{"key":"10.1016\/j.gene.2013.03.092_bb0195","doi-asserted-by":"crossref","first-page":"818","DOI":"10.1126\/science.1171242","article-title":"Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy","volume":"326","author":"Cartier","year":"2009","journal-title":"Science"},{"key":"10.1016\/j.gene.2013.03.092_bb0040","doi-asserted-by":"crossref","first-page":"5507","DOI":"10.1182\/blood-2010-05-283523","article-title":"High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors","volume":"116","author":"Cattoglio","year":"2010","journal-title":"Blood"},{"key":"10.1016\/j.gene.2013.03.092_bb0200","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. * Equal contribution","volume":"288","author":"Cavazzana-Calvo","year":"2000","journal-title":"Science"},{"key":"10.1016\/j.gene.2013.03.092_bb0050","doi-asserted-by":"crossref","first-page":"580","DOI":"10.1016\/j.coi.2012.08.008","article-title":"Gene therapy for primary immunodeficiencies: part 1","volume":"24","author":"Cavazzana-Calvo","year":"2012","journal-title":"Curr. Opin. Immunol."},{"key":"10.1016\/j.gene.2013.03.092_bb0055","doi-asserted-by":"crossref","first-page":"e1000491","DOI":"10.1371\/journal.pgen.1000491","article-title":"Murine leukemias with retroviral insertions at Lmo2 are predictive of the leukemias induced in SCID-X1 patients following retroviral gene therapy","volume":"5","author":"Dave","year":"2009","journal-title":"PLoS Genet."},{"key":"10.1016\/j.gene.2013.03.092_bb0060","doi-asserted-by":"crossref","first-page":"551","DOI":"10.1097\/ACI.0b013e32833fea85","article-title":"Update on gene therapy for adenosine deaminase-deficient severe combined immunodeficiency","volume":"10","author":"Ferrua","year":"2010","journal-title":"Curr. Opin. Allergy Clin. Immunol."},{"key":"10.1016\/j.gene.2013.03.092_bb0065","doi-asserted-by":"crossref","first-page":"237","DOI":"10.1016\/j.iac.2010.02.002","article-title":"Gene therapy for primary immunodeficiencies","volume":"30","author":"Fischer","year":"2010","journal-title":"Immunol. Allergy Clin. N. Am."},{"key":"10.1016\/j.gene.2013.03.092_bb0205","doi-asserted-by":"crossref","first-page":"97ra79","DOI":"10.1126\/scitranslmed.3002715","article-title":"Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency","volume":"3","author":"Gaspar","year":"2011","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.gene.2013.03.092_bb0210","doi-asserted-by":"crossref","first-page":"97ra80","DOI":"10.1126\/scitranslmed.3002716","article-title":"Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction","volume":"3","author":"Gaspar","year":"2011","journal-title":"Sci. Transl. Med."},{"issue":"602\u201310","key":"10.1016\/j.gene.2013.03.092_bb0080","first-page":"e1","article-title":"Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better?","volume":"126","author":"Gennery","year":"2010","journal-title":"J. Allergy Clin. Immunol."},{"key":"10.1016\/j.gene.2013.03.092_bb0085","doi-asserted-by":"crossref","first-page":"28","DOI":"10.1038\/mt.2010.232","article-title":"Gene therapy of chronic granulomatous disease: the engraftment dilemma","volume":"19","author":"Grez","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.gene.2013.03.092_bb0090","doi-asserted-by":"crossref","first-page":"3132","DOI":"10.1172\/JCI35700","article-title":"Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1","volume":"118","author":"Hacein-Bey-Abina","year":"2008","journal-title":"J. Clin. Invest."},{"key":"10.1016\/j.gene.2013.03.092_bb0095","doi-asserted-by":"crossref","first-page":"355","DOI":"10.1056\/NEJMoa1000164","article-title":"Efficacy of gene therapy for X-linked severe combined immunodeficiency","volume":"363","author":"Hacein-Bey-Abina","year":"2010","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.gene.2013.03.092_bb0100","doi-asserted-by":"crossref","first-page":"731","DOI":"10.1038\/nbt.1927","article-title":"Genetic engineering of human pluripotent cells using TALE nucleases","volume":"29","author":"Hockemeyer","year":"2011","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.gene.2013.03.092_bb0105","doi-asserted-by":"crossref","first-page":"3143","DOI":"10.1172\/JCI35798","article-title":"Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients","volume":"118","author":"Howe","year":"2008","journal-title":"J. Clin. Invest."},{"key":"10.1016\/j.gene.2013.03.092_bb0110","doi-asserted-by":"crossref","first-page":"113","DOI":"10.1038\/mt.2010.203","article-title":"Gene therapy of canine leukocyte adhesion deficiency using lentiviral vectors with human CD11b and CD18 promoters driving canine CD18 expression","volume":"19","author":"Hunter","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.gene.2013.03.092_bb0115","doi-asserted-by":"crossref","first-page":"269","DOI":"10.1016\/j.semcancer.2010.06.003","article-title":"The genomic risk of somatic gene therapy","volume":"20","author":"Kustikova","year":"2010","journal-title":"Semin. Cancer Biol."},{"key":"10.1016\/j.gene.2013.03.092_bb0120","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1182\/blood-2005-05-2032","article-title":"Long-term immune reconstitution in RAG-1-deficient mice treated by retroviral gene therapy: a balance between efficiency and toxicity","volume":"107","author":"Lagresle-Peyrou","year":"2006","journal-title":"Blood"},{"key":"10.1016\/j.gene.2013.03.092_bb0125","doi-asserted-by":"crossref","first-page":"1298","DOI":"10.1038\/nbt1353","article-title":"Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery","volume":"25","author":"Lombardo","year":"2007","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.gene.2013.03.092_bb0130","doi-asserted-by":"crossref","first-page":"1409","DOI":"10.1084\/jem.20120846","article-title":"Thymus-autonomous T cell development in the absence of progenitor import","volume":"209","author":"Martins","year":"2012","journal-title":"J. Exp. Med."},{"key":"10.1016\/j.gene.2013.03.092_bb0135","doi-asserted-by":"crossref","first-page":"343","DOI":"10.1089\/hum.2010.060","article-title":"Large-scale manufacture and characterization of a lentiviral vector produced for clinical ex vivo gene therapy application","volume":"22","author":"Merten","year":"2011","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.gene.2013.03.092_bb0140","doi-asserted-by":"crossref","first-page":"1919","DOI":"10.1038\/mt.2009.179","article-title":"Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors","volume":"17","author":"Modlich","year":"2009","journal-title":"Mol. Ther."},{"key":"10.1016\/j.gene.2013.03.092_bb0145","doi-asserted-by":"crossref","first-page":"16406","DOI":"10.1073\/pnas.0608130103","article-title":"Complete correction of murine Artemis immunodeficiency by lentiviral vector-mediated gene transfer","volume":"103","author":"Mostoslavsky","year":"2006","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.gene.2013.03.092_bb0150","doi-asserted-by":"crossref","first-page":"1161","DOI":"10.1016\/j.jaci.2009.10.013","article-title":"Primary immunodeficiencies: 2009 update","volume":"124","author":"Notarangelo","year":"2009","journal-title":"J. Allergy Clin. Immunol."},{"key":"10.1016\/j.gene.2013.03.092_bb0155","doi-asserted-by":"crossref","first-page":"73","DOI":"10.1038\/nbt.1717","article-title":"Genomic safe harbors permit high beta-globin transgene expression in thalassemia induced pluripotent stem cells","volume":"29","author":"Papapetrou","year":"2011","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.gene.2013.03.092_bb0160","doi-asserted-by":"crossref","first-page":"1401","DOI":"10.1084\/jem.20120845","article-title":"Thymocytes may persist and differentiate without any input from bone marrow progenitors","volume":"209","author":"Peaudecerf","year":"2012","journal-title":"J. Exp. Med."},{"key":"10.1016\/j.gene.2013.03.092_bb0165","doi-asserted-by":"crossref","first-page":"e1001008","DOI":"10.1371\/journal.pcbi.1001008","article-title":"Deciphering the code for retroviral integration target site selection","volume":"6","author":"Santoni","year":"2010","journal-title":"PLoS Comput. Biol."},{"key":"10.1016\/j.gene.2013.03.092_bb0170","doi-asserted-by":"crossref","first-page":"521","DOI":"10.1038\/nature09591","article-title":"Direct conversion of human fibroblasts to multilineage blood progenitors","volume":"468","author":"Szabo","year":"2010","journal-title":"Nature"},{"key":"10.1016\/j.gene.2013.03.092_bb0175","doi-asserted-by":"crossref","first-page":"4255","DOI":"10.1182\/blood-2004-12-4837","article-title":"Failure of SCID-X1 gene therapy in older patients","volume":"105","author":"Thrasher","year":"2005","journal-title":"Blood"},{"key":"10.1016\/j.gene.2013.03.092_bb0180","doi-asserted-by":"crossref","first-page":"711","DOI":"10.1146\/annurev.biochem.74.050304.091637","article-title":"Gene therapy: twenty-first century medicine","volume":"74","author":"Verma","year":"2005","journal-title":"Annu. Rev. Biochem."},{"key":"10.1016\/j.gene.2013.03.092_bb0185","doi-asserted-by":"crossref","first-page":"3194","DOI":"10.1073\/pnas.83.10.3194","article-title":"Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells","volume":"83","author":"Yu","year":"1986","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.gene.2013.03.092_bb0190","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1038\/nbt.1775","article-title":"Efficient construction of sequence-specific TAL effectors for modulating mammalian transcription","volume":"29","author":"Zhang","year":"2011","journal-title":"Nat. Biotechnol."}],"container-title":["Gene"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0378111913003612?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0378111913003612?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2018,10,16]],"date-time":"2018-10-16T21:25:49Z","timestamp":1539725149000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0378111913003612"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2013,8]]},"references-count":38,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2013,8]]}},"alternative-id":["S0378111913003612"],"URL":"https:\/\/doi.org\/10.1016\/j.gene.2013.03.092","relation":{},"ISSN":["0378-1119"],"issn-type":[{"value":"0378-1119","type":"print"}],"subject":[],"published":{"date-parts":[[2013,8]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Gene therapy of primary T cell immunodeficiencies","name":"articletitle","label":"Article Title"},{"value":"Gene","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.gene.2013.03.092","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"Copyright \u00a9 2013 Elsevier B.V. All rights reserved.","name":"copyright","label":"Copyright"}]}}