{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,10]],"date-time":"2026-10-10T15:58:25Z","timestamp":1791647905276,"version":"4.3.4"},"reference-count":54,"publisher":"Elsevier BV","issue":"3","license":[{"start":{"date-parts":[[2014,3,1]],"date-time":"2014-03-01T00:00:00Z","timestamp":1393632000000},"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":951,"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":[[2014,3]]},"DOI":"10.1038\/mt.2013.265","type":"journal-article","created":{"date-parts":[[2013,11,20]],"date-time":"2013-11-20T07:25:28Z","timestamp":1384932328000},"page":"607-622","source":"Crossref","is-referenced-by-count":78,"title":["Preclinical Demonstration of Lentiviral Vector-mediated Correction of Immunological and Metabolic Abnormalities in Models of Adenosine Deaminase Deficiency"],"prefix":"10.1016","volume":"22","author":[{"given":"Denise A","family":"Carbonaro","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lin","family":"Zhang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiangyang","family":"Jin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudia","family":"Montiel-Equihua","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sabine","family":"Geiger","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marlene","family":"Carmo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aaron","family":"Cooper","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lynette","family":"Fairbanks","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael L","family":"Kaufman","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Neil J","family":"Sebire","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roger P","family":"Hollis","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael P","family":"Blundell","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shantha","family":"Senadheera","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pei-Yu","family":"Fu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Arineh","family":"Sahaghian","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rebecca Y","family":"Chan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaoyan","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kenneth","family":"Cornetta","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adrian J","family":"Thrasher","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Donald B","family":"Kohn","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"H Bobby","family":"Gaspar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1038\/mt.2013.265_bib1","first-page":"291","article-title":"Adenosine deaminase deficiency: clinical expression, molecular basis, and therapy","volume":"35","author":"Hershfield","year":"1998","journal-title":"Semin Hematol"},{"key":"10.1038\/mt.2013.265_bib2","doi-asserted-by":"crossref","first-page":"131","DOI":"10.1172\/JCI200110360","article-title":"Adenosine deaminase deficiency increases thymic apoptosis and causes defective T cell receptor signaling","volume":"108","author":"Apasov","year":"2001","journal-title":"J Clin Invest"},{"key":"10.1038\/mt.2013.265_bib3","doi-asserted-by":"crossref","first-page":"278","DOI":"10.1016\/j.jpeds.2003.10.055","article-title":"Bilateral sensorineural deafness in adenosine deaminase-deficient severe combined immunodeficiency","volume":"144","author":"Albuquerque","year":"2004","journal-title":"J Pediatr"},{"key":"10.1038\/mt.2013.265_bib4","first-page":"157","article-title":"Pathologic findings in adenosine deaminase-deficient severe combined immunodeficiency. I. Kidney, adrenal, and chondro-osseous tissue alterations","volume":"120","author":"Ratech","year":"1985","journal-title":"Am J Pathol"},{"key":"10.1038\/mt.2013.265_bib5","doi-asserted-by":"crossref","first-page":"5093","DOI":"10.1074\/jbc.273.9.5093","article-title":"Adenosine deaminase-deficient mice generated using a two-stage genetic engineering strategy exhibit a combined immunodeficiency","volume":"273","author":"Blackburn","year":"1998","journal-title":"J Biol Chem"},{"key":"10.1038\/mt.2013.265_bib6","doi-asserted-by":"crossref","first-page":"139","DOI":"10.1016\/j.clim.2006.12.009","article-title":"Management options for adenosine deaminase deficiency; proceedings of the EBMT satellite workshop (Hamburg, March 2006)","volume":"123","author":"Booth","year":"2007","journal-title":"Clin Immunol"},{"key":"10.1038\/mt.2013.265_bib7","doi-asserted-by":"crossref","first-page":"3524","DOI":"10.1182\/blood-2009-06-189209","article-title":"How I treat ADA deficiency","volume":"114","author":"Gaspar","year":"2009","journal-title":"Blood"},{"key":"10.1038\/mt.2013.265_bib8","doi-asserted-by":"crossref","first-page":"475","DOI":"10.1126\/science.270.5235.475","article-title":"T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years","volume":"270","author":"Blaese","year":"1995","journal-title":"Science"},{"key":"10.1038\/mt.2013.265_bib9","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":"10.1038\/mt.2013.265_bib10","doi-asserted-by":"crossref","first-page":"423","DOI":"10.1038\/nm0502-423","article-title":"Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement","volume":"8","author":"Aiuti","year":"2002","journal-title":"Nat Med"},{"key":"10.1038\/mt.2013.265_bib11","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":"10.1038\/mt.2013.265_bib12","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":"10.1038\/mt.2013.265_bib13","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"},{"key":"10.1038\/mt.2013.265_bib14","doi-asserted-by":"crossref","first-page":"1918","DOI":"10.1056\/NEJMoa1003548","article-title":"Stem-cell gene therapy for the Wiskott-Aldrich syndrome","volume":"363","author":"Boztug","year":"2010","journal-title":"N Engl J Med"},{"key":"10.1038\/mt.2013.265_bib15","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.2013.265_bib16","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":"10.1038\/mt.2013.265_bib17","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.2013.265_bib18","doi-asserted-by":"crossref","first-page":"401","DOI":"10.1038\/nm1393","article-title":"Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1","volume":"12","author":"Ott","year":"2006","journal-title":"Nat Med"},{"key":"10.1038\/mt.2013.265_bib19","doi-asserted-by":"crossref","first-page":"198","DOI":"10.1038\/nm.2088","article-title":"Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease","volume":"16","author":"Stein","year":"2010","journal-title":"Nat Med"},{"key":"10.1038\/mt.2013.265_bib20","doi-asserted-by":"crossref","first-page":"2233","DOI":"10.1172\/JCI31666","article-title":"Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy","volume":"117","author":"Aiuti","year":"2007","journal-title":"J Clin Invest"},{"key":"10.1038\/mt.2013.265_bib21","doi-asserted-by":"crossref","first-page":"8463","DOI":"10.1128\/JVI.72.11.8463-8471.1998","article-title":"A third-generation lentivirus vector with a conditional packaging system","volume":"72","author":"Dull","year":"1998","journal-title":"J Virol"},{"key":"10.1038\/mt.2013.265_bib22","doi-asserted-by":"crossref","first-page":"8150","DOI":"10.1128\/JVI.72.10.8150-8157.1998","article-title":"Development of a self-inactivating lentivirus vector","volume":"72","author":"Miyoshi","year":"1998","journal-title":"J Virol"},{"key":"10.1038\/mt.2013.265_bib23","doi-asserted-by":"crossref","first-page":"263","DOI":"10.1126\/science.272.5259.263","article-title":"In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector","volume":"272","author":"Naldini","year":"1996","journal-title":"Science"},{"key":"10.1038\/mt.2013.265_bib24","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":"10.1038\/mt.2013.265_bib25","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.2013.265_bib26","first-page":"255","article-title":"Gene therapy of x-linked adrenoleukodystrophy using hematopoietic stem cells and a lentiviral vector","volume":"194","author":"Cartier","year":"2010","journal-title":"Bull Acad Natl Med"},{"key":"10.1038\/mt.2013.265_bib27","doi-asserted-by":"crossref","first-page":"318","DOI":"10.1038\/nature09328","article-title":"Transfusion independence and HMGA2 activation after gene therapy of human \u00df-thalassaemia","volume":"467","author":"Cavazzana-Calvo","year":"2010","journal-title":"Nature"},{"key":"10.1038\/mt.2013.265_bib28","doi-asserted-by":"crossref","first-page":"1400","DOI":"10.1038\/mt.2012.50","article-title":"The beta-globin locus control region in combination with the EF1alpha short promoter allows enhanced lentiviral vector-mediated erythroid gene expression with conserved multilineage activity","volume":"20","author":"Montiel-Equihua","year":"2012","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2013.265_bib29","doi-asserted-by":"crossref","first-page":"1084","DOI":"10.1038\/mt.2012.93","article-title":"Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy","volume":"20","author":"Corrigan-Curay","year":"2012","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2013.265_bib30","doi-asserted-by":"crossref","first-page":"3677","DOI":"10.1182\/blood-2012-02-408591","article-title":"Gene therapy\/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction","volume":"120","author":"Carbonaro","year":"2012","journal-title":"Blood"},{"key":"10.1038\/mt.2013.265_bib31","doi-asserted-by":"crossref","first-page":"1149","DOI":"10.1172\/JCI9944","article-title":"Metabolites from apoptotic thymocytes inhibit thymopoiesis in adenosine deaminase-deficient fetal thymic organ cultures","volume":"106","author":"Thompson","year":"2000","journal-title":"J Clin Invest"},{"key":"10.1038\/mt.2013.265_bib32","doi-asserted-by":"crossref","first-page":"1640","DOI":"10.1038\/mt.2010.132","article-title":"A ubiquitous chromatin opening element (UCOE) confers resistance to DNA methylation-mediated silencing of lentiviral vectors","volume":"18","author":"Zhang","year":"2010","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2013.265_bib33","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.2013.265_bib34","doi-asserted-by":"crossref","first-page":"2979","DOI":"10.1182\/blood-2006-05-023507","article-title":"Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)-deficient mice and corrects their immune and metabolic defects","volume":"108","author":"Mortellaro","year":"2006","journal-title":"Blood"},{"key":"10.1038\/mt.2013.265_bib35","doi-asserted-by":"crossref","first-page":"449","DOI":"10.1007\/s10875-012-9658-3","article-title":"Non-infectious lung disease in patients with adenosine deaminase deficient severe combined immunodeficiency","volume":"32","author":"Booth","year":"2012","journal-title":"J Clin Immunol"},{"key":"10.1038\/mt.2013.265_bib36","doi-asserted-by":"crossref","first-page":"1096","DOI":"10.1016\/j.ymthe.2004.08.014","article-title":"IL-3 or IL-7 increases ex vivo gene transfer efficiency in ADA-SCID BM CD34+ cells while maintaining in vivo lymphoid potential","volume":"10","author":"Ficara","year":"2004","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2013.265_bib37","doi-asserted-by":"crossref","first-page":"1078","DOI":"10.1038\/gt.2011.63","article-title":"Optimal conditions for lentiviral transduction of engrafting human CD34+ cells","volume":"18","author":"Uchida","year":"2011","journal-title":"Gene Ther"},{"key":"10.1038\/mt.2013.265_bib38","doi-asserted-by":"crossref","first-page":"963","DOI":"10.1038\/ni.2405","article-title":"Lymphoid priming in human bone marrow begins before expression of CD10 with upregulation of L-selectin","volume":"13","author":"Kohn","year":"2012","journal-title":"Nat Immunol"},{"key":"10.1038\/mt.2013.265_bib39","doi-asserted-by":"crossref","first-page":"964","DOI":"10.1172\/JCI37630","article-title":"The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy","volume":"119","author":"Montini","year":"2009","journal-title":"J Clin Invest"},{"key":"10.1038\/mt.2013.265_bib40","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":"10.1038\/mt.2013.265_bib41","doi-asserted-by":"crossref","first-page":"1356","DOI":"10.1038\/sj.mt.6300159","article-title":"Comparison of HIV-derived lentiviral and MLV-based gammaretroviral vector integration sites in primate repopulating cells","volume":"15","author":"Beard","year":"2007","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2013.265_bib42","doi-asserted-by":"crossref","first-page":"423","DOI":"10.1089\/hum.2007.011","article-title":"Unique integration profiles in a canine model of long-term repopulating cells transduced with gammaretrovirus, lentivirus, or foamy virus","volume":"18","author":"Beard","year":"2007","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mt.2013.265_bib43","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1089\/humc.2013.119","article-title":"The committee for advanced therapies\u2019 of the European Medicines Agency reflection paper on management of clinical risks deriving from insertional mutagenesis","volume":"24","author":"Aiuti","year":"2013","journal-title":"Hum Gene Ther Clin Dev"},{"key":"10.1038\/mt.2013.265_bib44","doi-asserted-by":"crossref","first-page":"1233158","DOI":"10.1126\/science.1233158","article-title":"Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy","volume":"341","author":"Biffi","year":"2013","journal-title":"Science"},{"key":"10.1038\/mt.2013.265_bib45","doi-asserted-by":"crossref","first-page":"6477","DOI":"10.4049\/jimmunol.174.10.6477","article-title":"Human lymphoid and myeloid cell development in NOD\/LtSz-scid IL2R gamma null mice engrafted with mobilized human hemopoietic stem cells","volume":"174","author":"Shultz","year":"2005","journal-title":"J Immunol"},{"key":"10.1038\/mt.2013.265_bib46","doi-asserted-by":"crossref","first-page":"505","DOI":"10.1016\/j.ymthe.2006.06.007","article-title":"Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning","volume":"14","author":"Gaspar","year":"2006","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2013.265_bib47","doi-asserted-by":"crossref","first-page":"803","DOI":"10.1089\/10430340252898984","article-title":"High-level transduction and gene expression in hematopoietic repopulating cells using a human immunodeficiency [correction of imunodeficiency] virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter","volume":"13","author":"Demaison","year":"2002","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mt.2013.265_bib48","doi-asserted-by":"crossref","first-page":"435","DOI":"10.1006\/mthe.2000.0191","article-title":"Context dependence of different modules for posttranscriptional enhancement of gene expression from retroviral vectors","volume":"2","author":"Schambach","year":"2000","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2013.265_bib49","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.jviromet.2011.06.019","article-title":"Highly efficient large-scale lentiviral vector concentration by tandem tangential flow filtration","volume":"177","author":"Cooper","year":"2011","journal-title":"J Virol Methods"},{"key":"10.1038\/mt.2013.265_bib50","doi-asserted-by":"crossref","first-page":"89","DOI":"10.1016\/B978-0-12-386509-0.00005-3","article-title":"Developing novel lentiviral vectors into clinical products","volume":"507","author":"Leath","year":"2012","journal-title":"Meth Enzymol"},{"key":"10.1038\/mt.2013.265_bib51","doi-asserted-by":"crossref","first-page":"1121","DOI":"10.1016\/j.ymthe.2006.01.008","article-title":"Transient gene expression by nonintegrating lentiviral vectors","volume":"13","author":"Nightingale","year":"2006","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2013.265_bib52","doi-asserted-by":"crossref","first-page":"43","DOI":"10.1214\/aoms\/1177707036","article-title":"Variances of variance components: II. Unbalanced single classifications","volume":"28","author":"Tukey","year":"1957","journal-title":"The Annals of Mathematical Statistics"},{"key":"10.1038\/mt.2013.265_bib53","series-title":"Linear Mixed Models: A Practical Guide to Using Statistical Software","author":"West","year":"2007"},{"key":"10.1038\/mt.2013.265_bib54","unstructured":"SAS Institute (2012). SAS\/STAT user's guide, Version 9.3. 9.3 ed SAS Institute Cary, NC"}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001616311856?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001616311856?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2020,8,9]],"date-time":"2020-08-09T22:37:17Z","timestamp":1597012637000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001616311856"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2014,3]]},"references-count":54,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2014,3]]}},"alternative-id":["S1525001616311856"],"URL":"https:\/\/doi.org\/10.1038\/mt.2013.265","relation":{"has-review":[{"id-type":"doi","id":"10.3410\/f.718182856.793511909","asserted-by":"object"}]},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2014,3]]}}}