{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,5]],"date-time":"2026-10-05T18:36:52Z","timestamp":1791225412503,"version":"4.1.0"},"reference-count":37,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"6","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2011,12]]},"DOI":"10.1097\/aci.0b013e32834c230c","type":"journal-article","created":{"date-parts":[[2011,10,4]],"date-time":"2011-10-04T19:20:45Z","timestamp":1317756045000},"page":"545-550","source":"Crossref","is-referenced-by-count":40,"title":["Gene therapy for the Wiskott\u2013Aldrich syndrome"],"prefix":"10.1097","volume":"11","author":[{"given":"Anne","family":"Galy","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adrian J.","family":"Thrasher","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"276","reference":[{"key":"R1-9-20210128","doi-asserted-by":"crossref","first-page":"4010","DOI":"10.1182\/blood-2003-05-1592","article-title":"Mutations of the Wiskott-Aldrich syndrome protein (WASP): hotspots, effect on transcription, and translation and phenotypegenotype correlation.","volume":"104","author":"Jin","year":"2004","journal-title":"Blood"},{"key":"R2-9-20210128","doi-asserted-by":"crossref","first-page":"84","DOI":"10.1007\/s12026-008-8084-3","article-title":"Mutations of the Wiskott-Aldrich syndrome protein affect protein expression and dictate the clinical phenotypes.","volume":"44","author":"Ochs","year":"2009","journal-title":"Immunol Res"},{"key":"R3-9-20210128","doi-asserted-by":"crossref","first-page":"3797","DOI":"10.1182\/blood.V86.10.3797.bloodjournal86103797","article-title":"The Wiskott-Aldrich syndrome and X-linked congenital thrombocytopenia are caused by mutations of the same gene.","volume":"86","author":"Zhu","year":"1995","journal-title":"Blood"},{"key":"R4-9-20210128","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":"R6-9-20210128","first-page":"138","article-title":"New insights into the biology of Wiskott-Aldrich syndrome (WAS).","volume":"132-","author":"Thrasher","year":"2009","journal-title":"Hematol Am Soc Hematol Educ Program"},{"key":"R8-9-20210128","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1155\/2010\/781523","article-title":"The Wiskott-Aldrich syndrome: the actin cytoskeleton and immune cell function.","volume":"29","author":"Blundell","year":"2010","journal-title":"Dis Markers"},{"key":"R9-9-20210128","doi-asserted-by":"crossref","first-page":"182","DOI":"10.1038\/nri2724","article-title":"WASP: a key immunological multitasker.","volume":"10","author":"Thrasher","year":"2010","journal-title":"Nat Rev Immunol"},{"key":"R10-9-20210128","doi-asserted-by":"crossref","first-page":"778","DOI":"10.1016\/j.imbio.2009.06.009","article-title":"Wiskott-Aldrich syndrome: immunodeficiency resulting from defective cell migration and impaired immunostimulatory activation.","volume":"214","author":"Bouma","year":"2009","journal-title":"Immunobiology"},{"key":"R11-9-20210128","doi-asserted-by":"crossref","first-page":"68","DOI":"10.1097\/00000441-198708000-00002","article-title":"Bone marrow transplantation for immunodeficiency diseases.","volume":"294","author":"Good","year":"1987","journal-title":"Am J Med Sci"},{"key":"R12-9-20210128","doi-asserted-by":"crossref","first-page":"179","DOI":"10.1016\/j.iac.2010.02.001","article-title":"Hematopoietic cell transplantation for Wiskott-Aldrich syndrome: advances in biology and future directions for treatment.","volume":"30","author":"Pai","year":"2010","journal-title":"Immunol Allergy Clin North Am"},{"key":"R14-9-20210128","doi-asserted-by":"crossref","first-page":"127","DOI":"10.1007\/s12026-008-8091-4","article-title":"Revertant somatic mosaicism in the Wiskott-Aldrich syndrome.","volume":"44","author":"Davis","year":"2009","journal-title":"Immunol Res"},{"key":"R15-9-20210128","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 4-7, 2009.","volume":"27","author":"Stewart","year":"2007","journal-title":"J Clin Immunol"},{"key":"R16-9-20210128","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":"R17-9-20210128","doi-asserted-by":"crossref","first-page":"97ra79","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":"R18-9-20210128","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":"R19-9-20210128","doi-asserted-by":"crossref","first-page":"97ra80","DOI":"10.1126\/scitranslmed.3002715","article-title":"Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency.","volume":"3","author":"Gaspar","year":"2011","journal-title":"Sci Transl Med"},{"key":"R20-9-20210128","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":"R21-9-20210128","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":"R22-9-20210128","doi-asserted-by":"crossref","first-page":"12133","DOI":"10.1073\/pnas.94.22.12133","article-title":"Prolonged production of nadph oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease.","volume":"94","author":"Malech","year":"1997","journal-title":"Proc Natl Acad Sci U S A"},{"key":"R23-9-20210128","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":"R25-9-20210128","doi-asserted-by":"crossref","first-page":"783","DOI":"10.1182\/blood-2009-05-222760","article-title":"Retrovirus gene therapy for x-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils.","volume":"115","author":"Kang","year":"2010","journal-title":"Blood"},{"key":"R26-9-20210128","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":"R27-9-20210128","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":"R28-9-20210128","doi-asserted-by":"crossref","first-page":"75","DOI":"10.1002\/emmm.201000110","article-title":"Parachuting in the epigenome: the biology of gene vector insertion profiles in the context of clinical trials.","volume":"3","author":"Baum","year":"2011","journal-title":"EMBO Mol Med"},{"key":"R29-9-20210128","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":"R30-9-20210128","doi-asserted-by":"crossref","first-page":"1379","DOI":"10.1038\/nprot.2010.87","article-title":"Genome-wide high-throughput integrome analyses by nrLAM-PCR and next-generation sequencing.","volume":"5","author":"Paruzynski","year":"2010","journal-title":"Nat Protoc"},{"key":"R31-9-20210128","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":"R32-9-20210128","doi-asserted-by":"crossref","first-page":"649","DOI":"10.1089\/hum.2011.058","article-title":"Lentiviral vectors: their molecular design, safety, and use in laboratory and preclinical research.","volume":"22","author":"Dropulic","year":"2011","journal-title":"Hum Gene Ther"},{"key":"R33-9-20210128","doi-asserted-by":"crossref","first-page":"181","DOI":"10.1517\/14712598.8.2.181","article-title":"Development of lentiviral gene therapy for Wiskott-Aldrich syndrome.","volume":"8","author":"Galy","year":"2008","journal-title":"Expert Opin Biol Ther"},{"key":"R34-9-20210128","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":"R35-9-20210128","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":"R36-9-20210128","doi-asserted-by":"crossref","first-page":"5507","DOI":"10.1182\/blood-2010-05-283523","article-title":"High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors.","volume":"116","author":"Cattoglio","year":"2010","journal-title":"Blood"},{"key":"R37-9-20210128","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":"R38-9-20210128","doi-asserted-by":"crossref","first-page":"343","DOI":"10.1089\/hum.2010.060","article-title":"Large scale manufacture and characterisation of a lentiviral vector produced for clinical ex vivo gene therapy application.","volume":"22","author":"Merten","year":"2011","journal-title":"Hum Gene Ther"},{"key":"R39-9-20210128","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 promotercDNA-containing lentiviral vector and nonlethal irradiation.","volume":"17","author":"Dupre","year":"2006","journal-title":"Hum Gene Ther"},{"key":"R40-9-20210128","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":"R41-9-20210128","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"}],"container-title":["Current Opinion in Allergy & Clinical Immunology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.ovid.com\/10.1097\/ACI.0b013e32834c230c","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,8,6]],"date-time":"2026-08-06T20:20:16Z","timestamp":1786047616000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.ovid.com\/00130832-201112000-00009"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2011,12]]},"references-count":37,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2011]]}},"URL":"https:\/\/doi.org\/10.1097\/aci.0b013e32834c230c","relation":{},"ISSN":["1528-4050"],"issn-type":[{"value":"1528-4050","type":"print"}],"subject":[],"published":{"date-parts":[[2011,12]]}}}