{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,12]],"date-time":"2026-03-12T00:50:04Z","timestamp":1773276604625,"version":"3.50.1"},"reference-count":50,"publisher":"Elsevier BV","issue":"10","license":[{"start":{"date-parts":[[2011,10,1]],"date-time":"2011-10-01T00:00:00Z","timestamp":1317427200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2016,10,7]],"date-time":"2016-10-07T00:00:00Z","timestamp":1475798400000},"content-version":"vor","delay-in-days":1833,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2011,10]]},"DOI":"10.1038\/mt.2011.127","type":"journal-article","created":{"date-parts":[[2011,7,15]],"date-time":"2011-07-15T07:55:23Z","timestamp":1310716523000},"page":"1867-1877","source":"Crossref","is-referenced-by-count":39,"title":["Correction of Murine SCID-X1 by Lentiviral Gene Therapy Using a Codon-optimized IL2RG Gene and Minimal Pretransplant Conditioning"],"prefix":"10.1016","volume":"19","author":[{"given":"Marshall W","family":"Huston","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Niek P","family":"van Til","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Trudi P","family":"Visser","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shazia","family":"Arshad","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Martijn H","family":"Brugman","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Claudia","family":"Cattoglio","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ali","family":"Nowrouzi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yuedan","family":"Li","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Axel","family":"Schambach","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Manfred","family":"Schmidt","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Christopher","family":"Baum","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Christof","family":"von Kalle","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Fulvio","family":"Mavilio","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Fang","family":"Zhang","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mike P","family":"Blundell","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Adrian J","family":"Thrasher","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Monique MA","family":"Verstegen","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gerard","family":"Wagemaker","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1038\/mt.2011.127_bib1","doi-asserted-by":"crossref","first-page":"147","DOI":"10.1016\/0092-8674(93)90167-O","article-title":"Interleukin-2 receptor gamma chain mutation results in X-linked severe combined immunodeficiency in humans","volume":"73","author":"Noguchi","year":"1993","journal-title":"Cell"},{"key":"10.1038\/mt.2011.127_bib2","doi-asserted-by":"crossref","first-page":"1080","DOI":"10.1016\/S0140-6736(86)90477-0","article-title":"Bone-marrow transplantation for immunodeficiencies and osteopetrosis: European survey, 1968-1985","volume":"2","author":"Fischer","year":"1986","journal-title":"Lancet"},{"key":"10.1038\/mt.2011.127_bib3","doi-asserted-by":"crossref","first-page":"2181","DOI":"10.1016\/S0140-6736(04)17590-9","article-title":"Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector","volume":"364","author":"Gaspar","year":"2004","journal-title":"Lancet"},{"key":"10.1038\/mt.2011.127_bib4","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":"10.1038\/mt.2011.127_bib5","doi-asserted-by":"crossref","first-page":"2083","DOI":"10.1172\/JCI32949","article-title":"Retroviral integration and human gene therapy","volume":"117","author":"Bushman","year":"2007","journal-title":"J Clin Invest"},{"key":"10.1038\/mt.2011.127_bib6","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":"10.1038\/mt.2011.127_bib7","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":"10.1038\/mt.2011.127_bib8","doi-asserted-by":"crossref","first-page":"1820","DOI":"10.1128\/MCB.11.4.1820","article-title":"Retroviral insertions 90 kilobases proximal to the Evi-1 myeloid transforming gene activate transcription from the normal promoter","volume":"11","author":"Bartholomew","year":"1991","journal-title":"Mol Cell Biol"},{"key":"10.1038\/mt.2011.127_bib9","doi-asserted-by":"crossref","first-page":"2545","DOI":"10.1182\/blood-2005-08-024976","article-title":"Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity","volume":"108","author":"Modlich","year":"2006","journal-title":"Blood"},{"key":"10.1038\/mt.2011.127_bib10","doi-asserted-by":"crossref","first-page":"687","DOI":"10.1038\/nbt1216","article-title":"Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration","volume":"24","author":"Montini","year":"2006","journal-title":"Nat Biotechnol"},{"key":"10.1038\/mt.2011.127_bib11","doi-asserted-by":"crossref","first-page":"391","DOI":"10.1016\/j.ymthe.2005.08.012","article-title":"Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells","volume":"13","author":"Schambach","year":"2006","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2011.127_bib12","doi-asserted-by":"crossref","first-page":"9873","DOI":"10.1128\/JVI.72.12.9873-9880.1998","article-title":"Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery","volume":"72","author":"Zufferey","year":"1998","journal-title":"J Virol"},{"key":"10.1038\/mt.2011.127_bib13","doi-asserted-by":"crossref","first-page":"2140","DOI":"10.1073\/pnas.251682798","article-title":"Transgenesis by lentiviral vectors: lack of gene silencing in mammalian embryonic stem cells and preimplantation embryos","volume":"99","author":"Pfeifer","year":"2002","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2011.127_bib14","doi-asserted-by":"crossref","first-page":"10991","DOI":"10.1128\/JVI.75.22.10991-11001.2001","article-title":"Multiple effects of codon usage optimization on expression and immunogenicity of DNA candidate vaccines encoding the human immunodeficiency virus type 1 Gag protein","volume":"75","author":"Deml","year":"2001","journal-title":"J Virol"},{"key":"10.1038\/mt.2011.127_bib15","doi-asserted-by":"crossref","first-page":"111","DOI":"10.1038\/gt.2008.143","article-title":"Transgene optimization significantly improves SIN vector titers, gp91phox expression and reconstitution of superoxide production in X-CGD cells","volume":"16","author":"Moreno-Carranza","year":"2009","journal-title":"Gene Ther"},{"key":"10.1038\/mt.2011.127_bib16","doi-asserted-by":"crossref","first-page":"233","DOI":"10.1016\/0378-1119(93)90424-2","article-title":"An engineered PGK promoter and lac operator-repressor system for the regulation of gene expression in mammalian cells","volume":"130","author":"Hannan","year":"1993","journal-title":"Gene"},{"key":"10.1038\/mt.2011.127_bib17","doi-asserted-by":"crossref","first-page":"14849","DOI":"10.1074\/jbc.271.25.14849","article-title":"Tissue-specific activity of the gammac chain gene promoter depends upon an Ets binding site and is regulated by GA-binding protein","volume":"271","author":"Markiewicz","year":"1996","journal-title":"J Biol Chem"},{"key":"10.1038\/mt.2011.127_bib18","doi-asserted-by":"crossref","first-page":"718","DOI":"10.1038\/mt.2008.5","article-title":"Physiological promoters reduce the genotoxic risk of integrating gene vectors","volume":"16","author":"Zychlinski","year":"2008","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2011.127_bib19","first-page":"259","article-title":"HIV-based vectors. Preparation and use","volume":"69","author":"Follenzi","year":"2002","journal-title":"Methods Mol Med"},{"key":"10.1038\/mt.2011.127_bib20","doi-asserted-by":"crossref","first-page":"5329","DOI":"10.1182\/blood-2009-11-252874","article-title":"Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype","volume":"115","author":"van Til","year":"2010","journal-title":"Blood"},{"key":"10.1038\/mt.2011.127_bib21","doi-asserted-by":"crossref","first-page":"7541","DOI":"10.1128\/JVI.69.12.7541-7547.1995","article-title":"Novel retroviral vectors for efficient expression of the multidrug resistance (mdr-1) gene in early hematopoietic cells","volume":"69","author":"Baum","year":"1995","journal-title":"J Virol"},{"issue":"Database issue","key":"10.1038\/mt.2011.127_bib22","doi-asserted-by":"crossref","first-page":"D523","DOI":"10.1093\/nar\/gkh013","article-title":"RTCGD: retroviral tagged cancer gene database","volume":"32","author":"Akagi","year":"2004","journal-title":"Nucleic Acids Res"},{"key":"10.1038\/mt.2011.127_bib23","doi-asserted-by":"crossref","first-page":"241","DOI":"10.1016\/0022-1759(84)90291-6","article-title":"Measurement of primary in vivo IgM- and IgG-antibody response to KLH in humans: implications of pre-immune IgM binding in antigen-specific ELISA","volume":"74","author":"Korver","year":"1984","journal-title":"J Immunol Methods"},{"key":"10.1038\/mt.2011.127_bib24","article-title":"Process for determining the quantity of a DNA fragment of interest by a method of enzymatic amplification of DNA","author":"Pannetier","year":"1998","journal-title":"US patent 5,635,354"},{"key":"10.1038\/mt.2011.127_bib25","doi-asserted-by":"crossref","first-page":"4319","DOI":"10.1073\/pnas.90.9.4319","article-title":"The sizes of the CDR3 hypervariable regions of the murine T-cell receptor beta chains vary as a function of the recombined germ-line segments","volume":"90","author":"Pannetier","year":"1993","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2011.127_bib26","doi-asserted-by":"crossref","first-page":"1448","DOI":"10.1182\/blood-2006-12-060814","article-title":"Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells","volume":"110","author":"Zhang","year":"2007","journal-title":"Blood"},{"key":"10.1038\/mt.2011.127_bib27","doi-asserted-by":"crossref","first-page":"3027","DOI":"10.1182\/blood.V94.9.3027","article-title":"Restoration of lymphoid populations in a murine model of X-linked severe combined immunodeficiency by a gene-therapy approach","volume":"94","author":"Lo","year":"1999","journal-title":"Blood"},{"key":"10.1038\/mt.2011.127_bib28","doi-asserted-by":"crossref","first-page":"248","DOI":"10.1097\/00007890-198609000-00004","article-title":"Cure of murine thalassemia by bone marrow transplantation without eradication of endogenous stem cells","volume":"42","author":"Wagemaker","year":"1986","journal-title":"Transplantation"},{"key":"10.1038\/mt.2011.127_bib29","doi-asserted-by":"crossref","first-page":"900","DOI":"10.1182\/blood-2009-10-250209","article-title":"A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells","volume":"116","author":"Zhou","year":"2010","journal-title":"Blood"},{"key":"10.1038\/mt.2011.127_bib30","doi-asserted-by":"crossref","first-page":"2567","DOI":"10.1073\/pnas.91.7.2567","article-title":"Lack of expression from a retroviral vector after transduction of murine hematopoietic stem cells is associated with methylation in vivo","volume":"91","author":"Challita","year":"1994","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2011.127_bib31","doi-asserted-by":"crossref","first-page":"904","DOI":"10.1128\/JVI.65.2.904-912.1991","article-title":"Inactivation of the Moloney murine leukemia virus long terminal repeat in murine fibroblast cell lines is associated with methylation and dependent on its chromosomal position","volume":"65","author":"Hoeben","year":"1991","journal-title":"J Virol"},{"key":"10.1038\/mt.2011.127_bib32","doi-asserted-by":"crossref","first-page":"894","DOI":"10.1182\/blood.V96.3.894","article-title":"Inactivation of a GFP retrovirus occurs at multiple levels in long-term repopulating stem cells and their differentiated progeny","volume":"96","author":"Klug","year":"2000","journal-title":"Blood"},{"key":"10.1038\/mt.2011.127_bib33","doi-asserted-by":"crossref","first-page":"2852","DOI":"10.1101\/gad.12.18.2852","article-title":"Loss of transcriptional activity of a transgene is accompanied by DNA methylation and histone deacetylation and is prevented by insulators","volume":"12","author":"Pikaart","year":"1998","journal-title":"Genes Dev"},{"key":"10.1038\/mt.2011.127_bib34","doi-asserted-by":"crossref","first-page":"1171","DOI":"10.1126\/science.1105063","article-title":"Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking","volume":"308","author":"Kustikova","year":"2005","journal-title":"Science"},{"key":"10.1038\/mt.2011.127_bib35","doi-asserted-by":"crossref","first-page":"1123","DOI":"10.1038\/4401123a","article-title":"Gene therapy: therapeutic gene causing lymphoma","volume":"440","author":"Woods","year":"2006","journal-title":"Nature"},{"key":"10.1038\/mt.2011.127_bib36","doi-asserted-by":"crossref","first-page":"7","DOI":"10.1038\/nature05218","article-title":"Gene therapy: is IL2RG oncogenic in T-cell development","volume":"443","author":"Pike-Overzet","year":"2006","journal-title":"Nature"},{"key":"10.1038\/mt.2011.127_bib37","doi-asserted-by":"crossref","DOI":"10.1038\/nature05219","article-title":"Gene therapy: X-SCID transgene leukaemogenicity","volume":"443","author":"Thrasher","year":"2006","journal-title":"Nature"},{"key":"10.1038\/mt.2011.127_bib38","doi-asserted-by":"crossref","first-page":"754","DOI":"10.1038\/sj.leu.2404563","article-title":"Ectopic retroviral expression of LMO2, but not IL2Rgamma, blocks human T-cell development from CD34+ cells: implications for leukemogenesis in gene therapy","volume":"21","author":"Pike-Overzet","year":"2007","journal-title":"Leukemia"},{"key":"10.1038\/mt.2011.127_bib39","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":"Dav\u00e9","year":"2009","journal-title":"PLoS Genet"},{"key":"10.1038\/mt.2011.127_bib40","doi-asserted-by":"crossref","first-page":"1031","DOI":"10.1038\/mt.2009.59","article-title":"A novel model of SCID-X1 reconstitution reveals predisposition to retrovirus-induced lymphoma but no evidence of gammaC gene oncogenicity","volume":"17","author":"Scobie","year":"2009","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2011.127_bib41","doi-asserted-by":"crossref","first-page":"965","DOI":"10.1038\/mt.2010.50","article-title":"Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and gammac overexpression","volume":"18","author":"Ginn","year":"2010","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2011.127_bib42","first-page":"31","article-title":"HIV-1-derived lentiviral vectors","volume":"261","author":"Ailles","year":"2002","journal-title":"Curr Top Microbiol Immunol"},{"key":"10.1038\/mt.2011.127_bib43","doi-asserted-by":"crossref","first-page":"454","DOI":"10.1016\/S0076-6879(02)46071-5","article-title":"Generation of HIV-1 derived lentiviral vectors","volume":"346","author":"Follenzi","year":"2002","journal-title":"Meth Enzymol"},{"key":"10.1038\/mt.2011.127_bib44","doi-asserted-by":"crossref","first-page":"3886","DOI":"10.1182\/blood-2004-02-0656","article-title":"Monitoring the effect of gene silencing by RNA interference in human CD34+ cells injected into newborn RAG2-\/- gammac-\/- mice: functional inactivation of p53 in developing T cells","volume":"104","author":"Gimeno","year":"2004","journal-title":"Blood"},{"key":"10.1038\/mt.2011.127_bib45","doi-asserted-by":"crossref","first-page":"26","DOI":"10.1016\/j.ymthe.2004.09.012","article-title":"Kupffer cells and not liver sinusoidal endothelial cells prevent lentiviral transduction of hepatocytes","volume":"11","author":"van Til","year":"2005","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2011.127_bib46","doi-asserted-by":"crossref","first-page":"1161","DOI":"10.3181\/0802-RM-35","article-title":"PCR-based methodology for molecular microchimerism detection and quantification","volume":"233","author":"Pujal","year":"2008","journal-title":"Exp Biol Med (Maywood)"},{"key":"10.1038\/mt.2011.127_bib47","doi-asserted-by":"crossref","first-page":"1051","DOI":"10.1038\/nmeth1103","article-title":"High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR)","volume":"4","author":"Schmidt","year":"2007","journal-title":"Nat Methods"},{"key":"10.1038\/mt.2011.127_bib48","doi-asserted-by":"crossref","first-page":"373","DOI":"10.1007\/978-1-59745-409-4_25","article-title":"Retroviral insertion site analysis in dominant haematopoietic clones","volume":"506","author":"Kustikova","year":"2009","journal-title":"Methods Mol Biol"},{"key":"10.1038\/mt.2011.127_bib49","doi-asserted-by":"crossref","first-page":"810","DOI":"10.1126\/science.2814502","article-title":"Genomic sequencing and methylation analysis by ligation mediated PCR","volume":"246","author":"Pfeifer","year":"1989","journal-title":"Science"},{"key":"10.1038\/mt.2011.127_bib50","doi-asserted-by":"crossref","first-page":"2129","DOI":"10.1089\/104303400750001435","volume":"11","author":"Wognum","year":"2000","journal-title":"Hum Gene Ther"}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001616327794?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001616327794?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2020,6,21]],"date-time":"2020-06-21T09:31:05Z","timestamp":1592731865000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001616327794"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2011,10]]},"references-count":50,"journal-issue":{"issue":"10","published-print":{"date-parts":[[2011,10]]}},"alternative-id":["S1525001616327794"],"URL":"https:\/\/doi.org\/10.1038\/mt.2011.127","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2011,10]]}}}