{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,16]],"date-time":"2026-06-16T23:05:34Z","timestamp":1781651134579,"version":"3.54.5"},"reference-count":29,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2019,12,1]],"date-time":"2019-12-01T00:00:00Z","timestamp":1575158400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2019,12,1]],"date-time":"2019-12-01T00:00:00Z","timestamp":1575158400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2019,9,6]],"date-time":"2019-09-06T00:00:00Z","timestamp":1567728000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100013465","name":"French Muscular Dystrophy Association","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013465","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100007151","name":"Conseil D\u00c3\u00a9partemental de l'Essonne","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100007151","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy - Methods &amp; Clinical Development"],"published-print":{"date-parts":[[2019,12]]},"DOI":"10.1016\/j.omtm.2019.08.014","type":"journal-article","created":{"date-parts":[[2019,9,13]],"date-time":"2019-09-13T19:16:16Z","timestamp":1568402176000},"page":"232-245","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":22,"special_numbering":"C","title":["Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID"],"prefix":"10.1016","volume":"15","author":[{"given":"Sabine","family":"Charrier","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chantal","family":"Lagresle-Peyrou","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Valentina","family":"Poletti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6813-4705","authenticated-orcid":false,"given":"Michael","family":"Rothe","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gr\u00e9gory","family":"C\u00e9drone","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bernard","family":"Gjata","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0459-4320","authenticated-orcid":false,"given":"Fulvio","family":"Mavilio","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alain","family":"Fischer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Axel","family":"Schambach","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean-Pierre","family":"de Villartay","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marina","family":"Cavazzana","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Salima","family":"Hacein-Bey-Abina","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne","family":"Galy","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2019.08.014_bib1","doi-asserted-by":"crossref","first-page":"405","DOI":"10.1016\/j.jaci.2018.08.027","article-title":"The genetic landscape of severe combined immunodeficiency in the United States and Canada in the current era (2010-2018)","volume":"143","author":"Dvorak","year":"2019","journal-title":"J.\u00a0Allergy Clin. Immunol."},{"key":"10.1016\/j.omtm.2019.08.014_bib2","doi-asserted-by":"crossref","first-page":"177","DOI":"10.1016\/S0092-8674(01)00309-9","article-title":"Artemis, a novel DNA double-strand break repair\/V(D)J recombination protein, is mutated in human severe combined immune deficiency","volume":"105","author":"Moshous","year":"2001","journal-title":"Cell"},{"key":"10.1016\/j.omtm.2019.08.014_bib3","doi-asserted-by":"crossref","first-page":"315","DOI":"10.1084\/jem.20031142","article-title":"The etalol-beta-lactamase\/beta-CASP domain of Artemis constitutes the catalytic core for V(D)J recombination","volume":"199","author":"Poinsignon","year":"2004","journal-title":"J.\u00a0Exp. Med."},{"key":"10.1016\/j.omtm.2019.08.014_bib4","doi-asserted-by":"crossref","first-page":"27784","DOI":"10.1074\/jbc.M603047200","article-title":"Interplay between Ku, Artemis, and the DNA-dependent protein kinase catalytic subunit at DNA ends","volume":"281","author":"Drouet","year":"2006","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2019.08.014_bib5","doi-asserted-by":"crossref","first-page":"e1007541","DOI":"10.1371\/journal.pgen.1007541","article-title":"DNA replication stress triggers rapid DNA replication fork breakage by Artemis and XPF","volume":"14","author":"B\u00e9tous","year":"2018","journal-title":"PloS Genet."},{"key":"10.1016\/j.omtm.2019.08.014_bib6","doi-asserted-by":"crossref","first-page":"281","DOI":"10.1182\/blood-2013-01-476432","article-title":"SCID patients with ARTEMIS vs RAG deficiencies following HCT: increased risk of late toxicity in ARTEMIS-deficient SCID","volume":"123","author":"Schuetz","year":"2014","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2019.08.014_bib7","doi-asserted-by":"crossref","first-page":"4114","DOI":"10.1182\/blood-2008-09-177923","article-title":"Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency","volume":"113","author":"Neven","year":"2009","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2019.08.014_bib8","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.omtm.2019.08.014_bib9","doi-asserted-by":"crossref","first-page":"396","DOI":"10.1038\/sj.mt.6300353","article-title":"Restoration of human B-cell differentiation into NOD-SCID mice engrafted with gene-corrected CD34+ cells isolated from Artemis or RAG1-deficient patients","volume":"16","author":"Lagresle-Peyrou","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2019.08.014_bib10","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. USA"},{"key":"10.1016\/j.omtm.2019.08.014_bib11","doi-asserted-by":"crossref","first-page":"232","DOI":"10.1089\/hum.2014.062","article-title":"Role of transgene regulation in ex\u00a0vivo lentiviral correction of artemis deficiency","volume":"26","author":"Multhaup","year":"2015","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2019.08.014_bib12","doi-asserted-by":"crossref","first-page":"112","DOI":"10.1089\/hum.2016.064","article-title":"Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency","volume":"28","author":"Punwani","year":"2017","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2019.08.014_bib13","doi-asserted-by":"crossref","first-page":"865","DOI":"10.1089\/hum.2009.162","article-title":"Cytotoxicity associated with artemis overexpression after lentiviral vector-mediated gene transfer","volume":"21","author":"Multhaup","year":"2010","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2019.08.014_bib14","doi-asserted-by":"crossref","first-page":"1506","DOI":"10.1038\/bjc.2012.443","article-title":"Elevated expression of artemis in human fibroblast cells is associated with cellular radiosensitivity and increased apoptosis","volume":"107","author":"Ulus-Senguloglu","year":"2012","journal-title":"Br. J. Cancer"},{"key":"10.1016\/j.omtm.2019.08.014_bib15","doi-asserted-by":"crossref","first-page":"751","DOI":"10.1089\/dna.2011.1244","article-title":"Characterization of the human artemis promoter by heterologous gene expression in\u00a0vitro and in\u00a0vivo","volume":"30","author":"Multhaup","year":"2011","journal-title":"DNA Cell Biol."},{"key":"10.1016\/j.omtm.2019.08.014_bib16","doi-asserted-by":"crossref","first-page":"605","DOI":"10.1038\/gt.2009.3","article-title":"Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS","volume":"16","author":"Zanta-Boussif","year":"2009","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2019.08.014_bib17","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.omtm.2019.08.014_bib18","doi-asserted-by":"crossref","first-page":"1073","DOI":"10.1038\/mt.2009.31","article-title":"Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models","volume":"17","author":"Marangoni","year":"2009","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2019.08.014_bib19","doi-asserted-by":"crossref","first-page":"e62333","DOI":"10.1371\/journal.pone.0062333","article-title":"Mouse transplant models for evaluating the oncogenic risk of a self-inactivating XSCID lentiviral vector","volume":"8","author":"Zhou","year":"2013","journal-title":"PloS ONE"},{"key":"10.1016\/j.omtm.2019.08.014_bib20","doi-asserted-by":"crossref","first-page":"597","DOI":"10.1038\/sj.gt.3302440","article-title":"A lentiviral vector encoding the human Wiskott-Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice","volume":"12","author":"Charrier","year":"2005","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2019.08.014_bib21","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\u00a0vivo gene therapy application","volume":"22","author":"Merten","year":"2011","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2019.08.014_bib22","doi-asserted-by":"crossref","first-page":"176","DOI":"10.1038\/gt.2015.95","article-title":"Lymphopoiesis in transgenic mice over-expressing Artemis","volume":"23","author":"Rivera-Munoz","year":"2016","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2019.08.014_bib23","doi-asserted-by":"crossref","first-page":"703","DOI":"10.1038\/mt.2011.19","article-title":"Lentiviral vector integration profiles differ in rodent postmitotic tissues","volume":"19","author":"Bartholomae","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2019.08.014_bib24","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1016\/j.omtm.2018.03.002","article-title":"Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency","volume":"9","author":"Poletti","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2019.08.014_bib25","doi-asserted-by":"crossref","first-page":"335ra57","DOI":"10.1126\/scitranslmed.aad8856","article-title":"Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency","volume":"8","author":"De Ravin","year":"2016","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtm.2019.08.014_bib26","doi-asserted-by":"crossref","first-page":"2420","DOI":"10.4049\/jimmunol.174.4.2420","article-title":"Targeted disruption of the Artemis murine counterpart results in SCID and defective V(D)J recombination that is partially corrected with bone marrow transplantation","volume":"174","author":"Li","year":"2005","journal-title":"J.\u00a0Immunol."},{"key":"10.1016\/j.omtm.2019.08.014_bib27","doi-asserted-by":"crossref","first-page":"1082","DOI":"10.1016\/j.jaci.2008.09.037","article-title":"Omenn syndrome: inflammation in leaky severe combined immunodeficiency","volume":"122","author":"Villa","year":"2008","journal-title":"J.\u00a0Allergy Clin. Immunol."},{"key":"10.1016\/j.omtm.2019.08.014_bib28","doi-asserted-by":"crossref","first-page":"246","DOI":"10.1016\/j.jaci.2017.10.030","article-title":"Type I IFN-related NETosis in ataxia telangiectasia and Artemis deficiency","volume":"142","author":"Gul","year":"2018","journal-title":"J.\u00a0Allergy Clin. Immunol."},{"key":"10.1016\/j.omtm.2019.08.014_bib29","doi-asserted-by":"crossref","first-page":"3601","DOI":"10.1182\/blood-2008-11-188383","article-title":"Reduced immunoglobulin class switch recombination in the absence of Artemis","volume":"114","author":"Rivera-Munoz","year":"2009","journal-title":"Blood"}],"container-title":["Molecular Therapy - Methods &amp; Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050119300944?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050119300944?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,6]],"date-time":"2025-11-06T09:22:53Z","timestamp":1762420973000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050119300944"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,12]]},"references-count":29,"alternative-id":["S2329050119300944"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2019.08.014","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2019,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2019.08.014","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2019","name":"copyright","label":"Copyright"}]}}