{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,12]],"date-time":"2026-03-12T00:29:06Z","timestamp":1773275346165,"version":"3.50.1"},"reference-count":49,"publisher":"Elsevier BV","issue":"6","license":[{"start":{"date-parts":[[2012,6,1]],"date-time":"2012-06-01T00:00:00Z","timestamp":1338508800000},"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":1589,"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":[[2012,6]]},"DOI":"10.1038\/mt.2011.282","type":"journal-article","created":{"date-parts":[[2012,1,3]],"date-time":"2012-01-03T13:28:56Z","timestamp":1325597336000},"page":"1270-1279","source":"Crossref","is-referenced-by-count":26,"title":["Foamy Virus Vector-mediated Gene Correction of a Mouse Model of Wiskott\u2013Aldrich Syndrome"],"prefix":"10.1016","volume":"20","author":[{"given":"Toru","family":"Uchiyama","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marsilio","family":"Adriani","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"G Jayashree","family":"Jagadeesh","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Adam","family":"Paine","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Fabio","family":"Candotti","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1038\/mt.2011.282_bib1","doi-asserted-by":"crossref","first-page":"725","DOI":"10.1016\/j.jaci.2006.02.005","article-title":"The Wiskott-Aldrich syndrome","volume":"117","author":"Ochs","year":"2006","journal-title":"J Allergy Clin Immunol"},{"key":"10.1038\/mt.2011.282_bib2","doi-asserted-by":"crossref","first-page":"635","DOI":"10.1016\/0092-8674(94)90528-2","article-title":"Isolation of a novel gene mutated in Wiskott-Aldrich syndrome","volume":"78","author":"Derry","year":"1994","journal-title":"Cell"},{"key":"10.1038\/mt.2011.282_bib3","doi-asserted-by":"crossref","first-page":"635","DOI":"10.1038\/nri884","article-title":"WASp in immune-system organization and function","volume":"2","author":"Thrasher","year":"2002","journal-title":"Nat Rev Immunol"},{"key":"10.1038\/mt.2011.282_bib4","doi-asserted-by":"crossref","first-page":"439","DOI":"10.1182\/blood-2007-03-076679","article-title":"Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation","volume":"111","author":"Ozsahin","year":"2008","journal-title":"Blood"},{"key":"10.1038\/mt.2011.282_bib5","doi-asserted-by":"crossref","first-page":"2159","DOI":"10.1182\/blood-2002-05-1423","article-title":"Gene therapy for Wiskott-Aldrich syndrome: rescue of T-cell signaling and amelioration of colitis upon transplantation of retrovirally transduced hematopoietic stem cells in mice","volume":"101","author":"Klein","year":"2003","journal-title":"Blood"},{"key":"10.1038\/mt.2011.282_bib6","doi-asserted-by":"crossref","first-page":"3108","DOI":"10.1182\/blood-2002-11-3489","article-title":"Defects in T-cell-mediated immunity to influenza virus in murine Wiskott-Aldrich syndrome are corrected by oncoretroviral vector-mediated gene transfer into repopulating hematopoietic cells","volume":"102","author":"Strom","year":"2003","journal-title":"Blood"},{"key":"10.1038\/mt.2011.282_bib7","doi-asserted-by":"crossref","first-page":"303","DOI":"10.1089\/hum.2006.17.303","article-title":"Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WAS promoter\/cDNA-containing lentiviral vector and nonlethal irradiation","volume":"17","author":"Dupr\u00e9","year":"2006","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mt.2011.282_bib8","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1038\/sj.gt.3302863","article-title":"Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients","volume":"14","author":"Charrier","year":"2007","journal-title":"Gene Ther"},{"key":"10.1038\/mt.2011.282_bib9","doi-asserted-by":"crossref","first-page":"6288","DOI":"10.1182\/blood-2008-12-115253","article-title":"Recent advances in understanding the pathophysiology of Wiskott-Aldrich syndrome","volume":"113","author":"Bosticardo","year":"2009","journal-title":"Blood"},{"key":"10.1038\/mt.2011.282_bib10","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.2011.282_bib11","first-page":"181","article-title":"Human infection with foamy viruses","volume":"277","author":"Heneine","year":"2003","journal-title":"Curr Top Microbiol Immunol"},{"key":"10.1038\/mt.2011.282_bib12","first-page":"161","article-title":"Replication of primate foamy viruses in natural and experimental hosts","volume":"277","author":"Falcone","year":"2003","journal-title":"Curr Top Microbiol Immunol"},{"key":"10.1038\/mt.2011.282_bib13","doi-asserted-by":"crossref","first-page":"604","DOI":"10.1182\/blood.V98.3.604","article-title":"Gene transfer into murine hematopoietic stem cells with helper-free foamy virus vectors","volume":"98","author":"Vassilopoulos","year":"2001","journal-title":"Blood"},{"key":"10.1038\/mt.2011.282_bib14","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1089\/10430340460732481","article-title":"Transduction of long-term and mobilized peripheral blood-derived NOD\/SCID repopulating cells by foamy virus vectors","volume":"15","author":"Josephson","year":"2004","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mt.2011.282_bib15","doi-asserted-by":"crossref","first-page":"8295","DOI":"10.1073\/pnas.122131099","article-title":"Transduction of human NOD\/SCID-repopulating cells with both lymphoid and myeloid potential by foamy virus vectors","volume":"99","author":"Josephson","year":"2002","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2011.282_bib16","doi-asserted-by":"crossref","first-page":"509","DOI":"10.1089\/104303403764539305","article-title":"Comparison of three retroviral vector systems for transduction of nonobese diabetic\/severe combined immunodeficiency mice repopulating human CD34+ cord blood cells","volume":"14","author":"Leurs","year":"2003","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mt.2011.282_bib17","doi-asserted-by":"crossref","first-page":"93","DOI":"10.1038\/nm1695","article-title":"Successful treatment of canine leukocyte adhesion deficiency by foamy virus vectors","volume":"14","author":"Bauer","year":"2008","journal-title":"Nat Med"},{"key":"10.1038\/mt.2011.282_bib18","doi-asserted-by":"crossref","first-page":"65","DOI":"10.1182\/blood-2006-04-016741","article-title":"Foamy-virus-mediated gene transfer to canine repopulating cells","volume":"109","author":"Kiem","year":"2007","journal-title":"Blood"},{"key":"10.1038\/mt.2011.282_bib19","doi-asserted-by":"crossref","first-page":"4458","DOI":"10.1182\/blood-2007-07-102947","article-title":"Overnight transduction with foamyviral vectors restores the long-term repopulating activity of Fancc-\/- stem cells","volume":"112","author":"Si","year":"2008","journal-title":"Blood"},{"key":"10.1038\/mt.2011.282_bib20","doi-asserted-by":"crossref","first-page":"1498","DOI":"10.1073\/pnas.0510046103","article-title":"Foamy virus vector integration sites in normal human cells","volume":"103","author":"Trobridge","year":"2006","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2011.282_bib21","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.2011.282_bib22","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1016\/S1074-7613(02)00360-6","article-title":"Wiskott-Aldrich syndrome protein regulates lipid raft dynamics during immunological synapse formation","volume":"17","author":"Dupr\u00e9","year":"2002","journal-title":"Immunity"},{"key":"10.1038\/mt.2011.282_bib23","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 B cell activation","volume":"9","author":"Snapper","year":"1998","journal-title":"Immunity"},{"key":"10.1038\/mt.2011.282_bib24","doi-asserted-by":"crossref","first-page":"1329","DOI":"10.1084\/jem.190.9.1329","article-title":"Antigen receptor-induced activation and cytoskeletal rearrangement are impaired in Wiskott-Aldrich syndrome protein-deficient lymphocytes","volume":"190","author":"Zhang","year":"1999","journal-title":"J Exp Med"},{"key":"10.1038\/mt.2011.282_bib25","doi-asserted-by":"crossref","first-page":"1144","DOI":"10.1182\/blood-2004-03-1003","article-title":"Wiskott-Aldrich syndrome protein deficiency leads to reduced B-cell adhesion, migration, and homing, and a delayed humoral immune response","volume":"105","author":"Westerberg","year":"2005","journal-title":"Blood"},{"key":"10.1038\/mt.2011.282_bib26","doi-asserted-by":"crossref","first-page":"993","DOI":"10.1189\/jlb.0804444","article-title":"WASP deficiency leads to global defects of directed leukocyte migration in vitro and in vivo","volume":"77","author":"Snapper","year":"2005","journal-title":"J Leukoc Biol"},{"key":"10.1038\/mt.2011.282_bib27","doi-asserted-by":"crossref","first-page":"506","DOI":"10.1038\/sj.embor.7400665","article-title":"Wiskott-Aldrich syndrome protein is involved in \u03b1IIb \u03b23-mediated cell adhesion","volume":"7","author":"Tsuboi","year":"2006","journal-title":"EMBO Rep"},{"key":"10.1038\/mt.2011.282_bib28","doi-asserted-by":"crossref","first-page":"9648","DOI":"10.1073\/pnas.96.17.9648","article-title":"Wiskott-Aldrich syndrome protein regulates podosomes in primary human macrophages","volume":"96","author":"Linder","year":"1999","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2011.282_bib29","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.2011.282_bib30","doi-asserted-by":"crossref","first-page":"7305","DOI":"10.1128\/JVI.71.10.7305-7311.1997","article-title":"Human foamy virus reverse transcription that occurs late in the viral replication cycle","volume":"71","author":"Moebes","year":"1997","journal-title":"J Virol"},{"key":"10.1038\/mt.2011.282_bib31","doi-asserted-by":"crossref","first-page":"1579","DOI":"10.1126\/science.271.5255.1579","article-title":"Human foamy virus replication: a pathway distinct from that of retroviruses and hepadnaviruses","volume":"271","author":"Yu","year":"1996","journal-title":"Science"},{"key":"10.1038\/mt.2011.282_bib32","doi-asserted-by":"crossref","first-page":"2327","DOI":"10.1128\/JVI.78.5.2327-2335.2004","article-title":"Cell cycle requirements for transduction by foamy virus vectors compared to those of oncovirus and lentivirus vectors","volume":"78","author":"Trobridge","year":"2004","journal-title":"J Virol"},{"key":"10.1038\/mt.2011.282_bib33","doi-asserted-by":"crossref","first-page":"645","DOI":"10.1002\/jgm.1346","article-title":"Diverse genomic integration of a lentiviral vector developed for the treatment of Wiskott-Aldrich syndrome","volume":"11","author":"Mantovani","year":"2009","journal-title":"J Gene Med"},{"key":"10.1038\/mt.2011.282_bib34","doi-asserted-by":"crossref","first-page":"301","DOI":"10.1128\/MCB.8.1.301","article-title":"Identification of a common ecotropic viral integration site, Evi-1, in the DNA of AKXD murine myeloid tumors","volume":"8","author":"Mucenski","year":"1988","journal-title":"Mol Cell Biol"},{"key":"10.1038\/mt.2011.282_bib35","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.2011.282_bib36","doi-asserted-by":"crossref","first-page":"4139","DOI":"10.1182\/blood-2008-02-140715","article-title":"WASP confers selective advantage for specific hematopoietic cell populations and serves a unique role in marginal zone B-cell homeostasis and function","volume":"112","author":"Westerberg","year":"2008","journal-title":"Blood"},{"key":"10.1038\/mt.2011.282_bib37","doi-asserted-by":"crossref","first-page":"4158","DOI":"10.1182\/blood-2008-02-140814","article-title":"Wiskott-Aldrich syndrome protein deficiency in B cells results in impaired peripheral homeostasis","volume":"112","author":"Meyer-Bahlburg","year":"2008","journal-title":"Blood"},{"key":"10.1038\/mt.2011.282_bib38","doi-asserted-by":"crossref","first-page":"676","DOI":"10.1182\/blood-2003-05-1739","article-title":"Differential contribution of Wiskott-Aldrich syndrome protein to selective advantage in T- and B-cell lineages","volume":"103","author":"Konno","year":"2004","journal-title":"Blood"},{"key":"10.1038\/mt.2011.282_bib39","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":"10.1038\/mt.2011.282_bib40","doi-asserted-by":"crossref","first-page":"836","DOI":"10.1038\/mt.2008.43","article-title":"Improvement of migratory defects in a murine model of Wiskott-Aldrich syndrome gene therapy","volume":"16","author":"Blundell","year":"2008","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2011.282_bib41","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":"10.1038\/mt.2011.282_bib42","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":"10.1038\/mt.2011.282_bib43","doi-asserted-by":"crossref","first-page":"715","DOI":"10.1038\/sj.gt.3302457","article-title":"Lentiviral vectors transcriptionally targeted to hematopoietic cells by WASP gene proximal promoter sequences","volume":"12","author":"Mart\u00edn","year":"2005","journal-title":"Gene Ther"},{"key":"10.1038\/mt.2011.282_bib44","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1186\/1472-6750-5-17","article-title":"CpG-island fragments from the HNRPA2B1\/CBX3 genomic locus reduce silencing and enhance transgene expression from the hCMV promoter\/enhancer in mammalian cells","volume":"5","author":"Williams","year":"2005","journal-title":"BMC Biotechnol"},{"key":"10.1038\/mt.2011.282_bib45","doi-asserted-by":"crossref","first-page":"269","DOI":"10.1016\/S0888-7543(03)00107-1","article-title":"Transgenes encompassing dual-promoter CpG islands from the human TBP and HNRPA2B1 loci are resistant to heterochromatin-mediated silencing","volume":"82","author":"Antoniou","year":"2003","journal-title":"Genomics"},{"key":"10.1038\/mt.2011.282_bib46","doi-asserted-by":"crossref","first-page":"1376","DOI":"10.1016\/j.jaci.2011.03.030","article-title":"Lentiviral-mediated gene therapy leads to improvement of B-cell functionality in a murine model of Wiskott-Aldrich syndrome","volume":"127","author":"Bosticardo","year":"2011","journal-title":"J Allergy Clin Immunol"},{"key":"10.1038\/mt.2011.282_bib47","doi-asserted-by":"crossref","first-page":"321","DOI":"10.1006\/mthe.2002.0672","article-title":"Improved foamy virus vectors with minimal viral sequences","volume":"6","author":"Trobridge","year":"2002","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2011.282_bib48","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":"10.1038\/mt.2011.282_bib49","doi-asserted-by":"crossref","first-page":"39474","DOI":"10.1074\/jbc.M504672200","article-title":"Rac1 is essential for platelet lamellipodia formation and aggregate stability under flow","volume":"280","author":"McCarty","year":"2005","journal-title":"J Biol Chem"}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001616325515?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001616325515?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2020,6,29]],"date-time":"2020-06-29T23:44:42Z","timestamp":1593474282000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001616325515"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2012,6]]},"references-count":49,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2012,6]]}},"alternative-id":["S1525001616325515"],"URL":"https:\/\/doi.org\/10.1038\/mt.2011.282","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2012,6]]}}}