{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,5]],"date-time":"2026-10-05T18:36:40Z","timestamp":1791225400766,"version":"4.1.0"},"reference-count":50,"publisher":"Elsevier BV","issue":"6","license":[{"start":{"date-parts":[[2009,6,1]],"date-time":"2009-06-01T00:00:00Z","timestamp":1243814400000},"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":2685,"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":[[2009,6]]},"DOI":"10.1038\/mt.2009.31","type":"journal-article","created":{"date-parts":[[2009,3,3]],"date-time":"2009-03-03T15:05:34Z","timestamp":1236092734000},"page":"1073-1082","source":"Crossref","is-referenced-by-count":69,"title":["Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott\u2013Aldrich Syndrome in Preclinical Models"],"prefix":"10.1016","volume":"17","author":[{"given":"Francesco","family":"Marangoni","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marita","family":"Bosticardo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sabine","family":"Charrier","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elena","family":"Draghici","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michela","family":"Locci","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Samantha","family":"Scaramuzza","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cristina","family":"Panaroni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maurilio","family":"Ponzoni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesca","family":"Sanvito","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudio","family":"Doglioni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marie","family":"Liabeuf","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bernard","family":"Gjata","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marie","family":"Montus","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katherine","family":"Siminovitch","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alessandro","family":"Aiuti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Luigi","family":"Naldini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lo\u00efc","family":"Dupr\u00e9","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria Grazia","family":"Roncarolo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne","family":"Galy","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Villa","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1038\/mt.2009.31_bib1","doi-asserted-by":"crossref","first-page":"485","DOI":"10.1097\/01.all.0000191243.25757.ce","article-title":"WASP and the phenotypic range associated with deficiency","volume":"5","author":"Notarangelo","year":"2005","journal-title":"Curr Opin Allergy Clin Immunol"},{"key":"10.1038\/mt.2009.31_bib2","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.2009.31_bib3","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.2009.31_bib4","doi-asserted-by":"crossref","first-page":"2627","DOI":"10.1172\/JCI118712","article-title":"Studies of the expression of the Wiskott-Aldrich syndrome protein","volume":"97","author":"Stewart","year":"1996","journal-title":"J Clin Invest"},{"key":"10.1038\/mt.2009.31_bib5","doi-asserted-by":"crossref","first-page":"151","DOI":"10.1038\/35004513","article-title":"Autoinhibition and activation mechanisms of the Wiskott-Aldrich syndrome protein","volume":"404","author":"Kim","year":"2000","journal-title":"Nature"},{"key":"10.1038\/mt.2009.31_bib6","doi-asserted-by":"crossref","first-page":"7451","DOI":"10.4049\/jimmunol.177.10.7451","article-title":"Defective Th1 cytokine gene transcription in CD4+ and CD8+ T Cells from Wiskott-Aldrich Syndrome patients","volume":"177","author":"Trifari","year":"2006","journal-title":"J Immunol"},{"key":"10.1038\/mt.2009.31_bib7","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.2009.31_bib8","doi-asserted-by":"crossref","first-page":"436","DOI":"10.1182\/blood-2003-07-2621","article-title":"Impaired natural and CD16-mediated NK cell cytotoxicity in patients with WAS and XLT: ability of IL-2 to correct NK cell functional defect","volume":"104","author":"Gismondi","year":"2004","journal-title":"Blood"},{"key":"10.1038\/mt.2009.31_bib9","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1016\/j.immuni.2006.06.014","article-title":"Impaired integrin-dependent function in Wiskott-Aldrich syndrome protein-deficient murine and human neutrophils","volume":"25","author":"Zhang","year":"2006","journal-title":"Immunity"},{"key":"10.1038\/mt.2009.31_bib10","doi-asserted-by":"crossref","first-page":"4278","DOI":"10.1182\/blood-2007-06-096875","article-title":"Impaired T-cell priming in vivo resulting from dysfunction of WASp-deficient dendritic cells","volume":"110","author":"Bouma","year":"2007","journal-title":"Blood"},{"key":"10.1038\/mt.2009.31_bib11","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.2009.31_bib12","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":"Dupre","year":"2002","journal-title":"Immunity"},{"key":"10.1038\/mt.2009.31_bib13","doi-asserted-by":"crossref","first-page":"903","DOI":"10.1016\/j.ymthe.2004.08.008","article-title":"Lentiviral vector-mediated gene transfer in T cells from Wiskott-Aldrich syndrome patients leads to functional correction","volume":"10","author":"Dupre","year":"2004","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2009.31_bib14","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.2009.31_bib15","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.2009.31_bib16","doi-asserted-by":"crossref","first-page":"1598","DOI":"10.1182\/blood.V97.6.1598","article-title":"Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program","volume":"97","author":"Filipovich","year":"2001","journal-title":"Blood"},{"key":"10.1038\/mt.2009.31_bib17","doi-asserted-by":"crossref","first-page":"362","DOI":"10.1111\/j.1365-2141.2006.06297.x","article-title":"Outcome in patients with Wiskott-Aldrich syndrome following stem cell transplantation: an analysis of 57 patients in Japan","volume":"135","author":"Kobayashi","year":"2006","journal-title":"Br J Haematol"},{"key":"10.1038\/mt.2009.31_bib18","doi-asserted-by":"crossref","first-page":"671","DOI":"10.1038\/sj.bmt.1705512","article-title":"Stem cell transplantation for the Wiskott-Aldrich syndrome: a single-center experience confirms efficacy of matched unrelated donor transplantation","volume":"38","author":"Pai","year":"2006","journal-title":"Bone Marrow Transplant"},{"key":"10.1038\/mt.2009.31_bib19","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.2009.31_bib20","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.2009.31_bib21","doi-asserted-by":"crossref","first-page":"1185","DOI":"10.1056\/NEJMoa012616","article-title":"Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy","volume":"346","author":"Hacein-Bey-Abina","year":"2002","journal-title":"N Engl J Med"},{"key":"10.1038\/mt.2009.31_bib22","doi-asserted-by":"crossref","first-page":"375","DOI":"10.1097\/MOH.0b013e328302c807","article-title":"Recent advances in gene therapy for severe congenital immunodeficiency diseases","volume":"15","author":"Sokolic","year":"2008","journal-title":"Curr Opin Hematol"},{"key":"10.1038\/mt.2009.31_bib23","doi-asserted-by":"crossref","first-page":"4257","DOI":"10.1182\/blood-2005-10-4047","article-title":"Proteasome activity restricts lentiviral gene transfer into hematopoietic stem cells and is down-regulated by cytokines that enhance transduction","volume":"107","author":"Santoni de Sio","year":"2006","journal-title":"Blood"},{"key":"10.1038\/mt.2009.31_bib24","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.2009.31_bib25","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.2009.31_bib26","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":"Dupre","year":"2006","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mt.2009.31_bib27","doi-asserted-by":"crossref","first-page":"1253","DOI":"10.1038\/3233","article-title":"Thymic lymphoproliferative disease after successful correction of CD40 ligand deficiency by gene transfer in mice","volume":"4","author":"Brown","year":"1998","journal-title":"Nat Med"},{"key":"10.1038\/mt.2009.31_bib28","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.2009.31_bib29","doi-asserted-by":"crossref","first-page":"497","DOI":"10.1126\/science.1068893","article-title":"Murine leukemia induced by retroviral gene marking","volume":"296","author":"Li","year":"2002","journal-title":"Science"},{"key":"10.1038\/mt.2009.31_bib30","doi-asserted-by":"crossref","first-page":"726","DOI":"10.4049\/jimmunol.173.2.726","article-title":"Selective requirement for the Wiskott-Aldrich syndrome protein in cytokine, but not chemokine, secretion by CD4+ T cells","volume":"173","author":"Morales-Tirado","year":"2004","journal-title":"J Immunol"},{"key":"10.1038\/mt.2009.31_bib31","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.2009.31_bib32","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.2009.31_bib33","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.2009.31_bib34","doi-asserted-by":"crossref","first-page":"4554","DOI":"10.1182\/blood.V91.12.4554","article-title":"A 5\u2032 regulatory sequence containing two Ets motifs controls the expression of the Wiskott-Aldrich Syndrome protein (WASP) gene in human hematopoietic cells","volume":"91","author":"Petrella","year":"1998","journal-title":"Blood"},{"key":"10.1038\/mt.2009.31_bib35","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":"Martin","year":"2005","journal-title":"Gene Ther"},{"key":"10.1038\/mt.2009.31_bib36","doi-asserted-by":"crossref","first-page":"179","DOI":"10.1089\/hum.2007.098","article-title":"Hematopoietic-specific lentiviral vectors circumvent cellular toxicity due to ectopic expression of Wiskott-Aldrich syndrome protein","volume":"19","author":"Toscano","year":"2008","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mt.2009.31_bib37","doi-asserted-by":"crossref","first-page":"926","DOI":"10.1073\/pnas.0610275104","article-title":"WIP is a chaperone for Wiskott-Aldrich syndrome protein (WASP)","volume":"104","author":"de la Fuente","year":"2007","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2009.31_bib38","doi-asserted-by":"crossref","first-page":"146","DOI":"10.3346\/jkms.2008.23.1.146","article-title":"Unrelated bone marrow transplantation with a reduced toxicity myeloablative conditioning regimen in Wiskott-Aldrich syndrome","volume":"23","author":"Kang","year":"2008","journal-title":"J Korean Med Sci"},{"key":"10.1038\/mt.2009.31_bib39","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.2009.31_bib40","doi-asserted-by":"crossref","first-page":"1208","DOI":"10.1182\/blood-2002-01-0211","article-title":"Mixed chimera status of 12 patients with Wiskott-Aldrich syndrome (WAS) after hematopoietic stem cell transplantation: evaluation by flow cytometric analysis of intracellular WAS protein expression","volume":"100","author":"Yamaguchi","year":"2002","journal-title":"Blood"},{"key":"10.1038\/mt.2009.31_bib41","doi-asserted-by":"crossref","first-page":"634","DOI":"10.1007\/s10875-007-9121-z","article-title":"The phenomenon of spontaneous genetic reversions in the Wiskott-Aldrich syndrome: a report of the workshop of the ESID Genetics Working Party at the XIIth Meeting of the European Society for Immunodeficiencies (ESID). Budapest, Hungary October 4\u20137, 2006","volume":"27","author":"Stewart","year":"2007","journal-title":"J Clin Immunol"},{"key":"10.1038\/mt.2009.31_bib42","doi-asserted-by":"crossref","first-page":"1119","DOI":"10.4049\/jimmunol.165.2.1119","article-title":"Determination of carrier status for the Wiskott-Aldrich syndrome by flow cytometric analysis of Wiskott-Aldrich syndrome protein expression in peripheral blood mononuclear cells","volume":"165","author":"Yamada","year":"2000","journal-title":"J Immunol"},{"key":"10.1038\/mt.2009.31_bib43","doi-asserted-by":"crossref","first-page":"3872","DOI":"10.1182\/blood.V94.11.3872","article-title":"Spontaneous apoptosis in lymphocytes from patients with Wiskott-Aldrich syndrome: correlation of accelerated cell death and attenuated bcl-2 expression","volume":"94","author":"Rawlings","year":"1999","journal-title":"Blood"},{"key":"10.1038\/mt.2009.31_bib44","doi-asserted-by":"crossref","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","author":"Zanta-Boussif","year":"2009","journal-title":"Gene Ther"},{"key":"10.1038\/mt.2009.31_bib45","doi-asserted-by":"crossref","first-page":"456","DOI":"10.1182\/blood-2003-05-1480","article-title":"Clinical course of patients with WASP gene mutations","volume":"103","author":"Imai","year":"2004","journal-title":"Blood"},{"key":"10.1038\/mt.2009.31_bib46","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.2009.31_bib47","doi-asserted-by":"crossref","first-page":"930","DOI":"10.1038\/gt.2008.20","article-title":"Improved lentiviral vectors for Wiskott-Aldrich syndrome gene therapy mimic endogenous expression profiles throughout haematopoiesis","volume":"15","author":"Frecha","year":"2008","journal-title":"Gene Ther"},{"key":"10.1038\/mt.2009.31_bib48","doi-asserted-by":"crossref","first-page":"782","DOI":"10.1038\/sj.mt.6300083","article-title":"Importance of murine study design for testing toxicity of retroviral vectors in support of phase I trials","volume":"15","author":"Will","year":"2007","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2009.31_bib49","doi-asserted-by":"crossref","first-page":"2213","DOI":"10.1084\/jem.20062324","article-title":"Unregulated actin polymerization by WASp causes defects of mitosis and cytokinesis in X-linked neutropenia","volume":"204","author":"Moulding","year":"2007","journal-title":"J Exp Med"},{"key":"10.1038\/mt.2009.31_bib50","doi-asserted-by":"crossref","first-page":"3","DOI":"10.1038\/sj.gt.3302417","article-title":"Potential oncogene activity of the woodchuck hepatitis post-transcriptional regulatory element (WPRE)","volume":"12","author":"Kingsman","year":"2005","journal-title":"Gene Ther"}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001616318135?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001616318135?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2020,5,16]],"date-time":"2020-05-16T08:09:56Z","timestamp":1589616596000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001616318135"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2009,6]]},"references-count":50,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2009,6]]}},"alternative-id":["S1525001616318135"],"URL":"https:\/\/doi.org\/10.1038\/mt.2009.31","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2009,6]]}}}