{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,25]],"date-time":"2026-10-25T09:02:57Z","timestamp":1792918977048,"version":"4.5.0"},"reference-count":59,"publisher":"American Association for the Advancement of Science (AAAS)","issue":"227","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Sci. Transl. Med."],"published-print":{"date-parts":[[2014,3,12]]},"abstract":"<jats:p>Wiskott-Aldrich syndrome gene therapy is feasible, but \u03b3-retroviral vectors contribute a substantial risk of leukemogenesis.<\/jats:p>","DOI":"10.1126\/scitranslmed.3007280","type":"journal-article","created":{"date-parts":[[2014,3,12]],"date-time":"2014-03-12T14:05:41Z","timestamp":1394633141000},"source":"Crossref","is-referenced-by-count":524,"title":["Gene Therapy for Wiskott-Aldrich Syndrome\u2014Long-Term Efficacy and Genotoxicity"],"prefix":"10.1126","volume":"6","author":[{"given":"Christian J\u00f6rg","family":"Braun","sequence":"first","affiliation":[{"name":"Dr. von Hauner Children\u2019s Hospital, Ludwig Maximilians University Munich, 80337 Munich, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kaan","family":"Boztug","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology\/Oncology, Hannover Medical School, 30625 Hannover, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Paruzynski","sequence":"additional","affiliation":[{"name":"Department of Translational Oncology, National Center for Tumor Diseases and German Cancer Research Center, 69120 Heidelberg, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maximilian","family":"Witzel","sequence":"additional","affiliation":[{"name":"Dr. von Hauner Children\u2019s Hospital, Ludwig Maximilians University Munich, 80337 Munich, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adrian","family":"Schwarzer","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology\/Oncology, Hannover Medical School, 30625 Hannover, Germany."},{"name":"Institute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Rothe","sequence":"additional","affiliation":[{"name":"Institute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ute","family":"Modlich","sequence":"additional","affiliation":[{"name":"Institute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rita","family":"Beier","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology\/Oncology, Hannover Medical School, 30625 Hannover, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gudrun","family":"G\u00f6hring","sequence":"additional","affiliation":[{"name":"Institute for Cell and Molecular Pathology, Hannover Medical School, 30625 Hannover, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Doris","family":"Steinemann","sequence":"additional","affiliation":[{"name":"Institute for Cell and Molecular Pathology, Hannover Medical School, 30625 Hannover, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Raffaele","family":"Fronza","sequence":"additional","affiliation":[{"name":"Department of Translational Oncology, National Center for Tumor Diseases and German Cancer Research Center, 69120 Heidelberg, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudia Regina","family":"Ball","sequence":"additional","affiliation":[{"name":"Department of Translational Oncology, National Center for Tumor Diseases and German Cancer Research Center, 69120 Heidelberg, Germany."},{"name":"German Cancer Consortium."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Reinhard","family":"Haemmerle","sequence":"additional","affiliation":[{"name":"Institute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sonja","family":"Naundorf","sequence":"additional","affiliation":[{"name":"EUFETS AG, 55743 Idar-Oberstein, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Klaus","family":"K\u00fchlcke","sequence":"additional","affiliation":[{"name":"EUFETS AG, 55743 Idar-Oberstein, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Martina","family":"Rose","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Minden Hospital, 32429 Minden, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chris","family":"Fraser","sequence":"additional","affiliation":[{"name":"Queensland Children\u2019s Cancer Centre, Royal Children\u2019s Hospital, Brisbane 4006, Queensland, Australia."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Liesl","family":"Mathias","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology and Oncology, Loma Linda University Medical Center, Loma Linda, CA 92354, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rudolf","family":"Ferrari","sequence":"additional","affiliation":[{"name":"Children\u2019s Hospital Kemperhof, 56073 Koblenz, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miguel R.","family":"Abboud","sequence":"additional","affiliation":[{"name":"Department of Pediatrics and Adolescent Medicine, American University of Beirut Medical Center, Beirut 1107 2020, Lebanon."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Waleed","family":"Al-Herz","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Faculty of Medicine, Kuwait University, Kuwait City, Kuwait."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Irina","family":"Kondratenko","sequence":"additional","affiliation":[{"name":"Department of Clinical Immunology, Russian Clinical Children\u2019s Hospital, 117513 Moscow, Russia."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"L\u00e1szl\u00f3","family":"Mar\u00f3di","sequence":"additional","affiliation":[{"name":"Department of Infectious and Pediatric Immunology, Medical and Health Science Center, University of Debrecen, 4032 Debrecen, Hungary."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hanno","family":"Glimm","sequence":"additional","affiliation":[{"name":"Department of Translational Oncology, National Center for Tumor Diseases and German Cancer Research Center, 69120 Heidelberg, Germany."},{"name":"German Cancer Consortium."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brigitte","family":"Schlegelberger","sequence":"additional","affiliation":[{"name":"Institute for Cell and Molecular Pathology, Hannover Medical School, 30625 Hannover, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Axel","family":"Schambach","sequence":"additional","affiliation":[{"name":"Institute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael Heinrich","family":"Albert","sequence":"additional","affiliation":[{"name":"Dr. von Hauner Children\u2019s Hospital, Ludwig Maximilians University Munich, 80337 Munich, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Manfred","family":"Schmidt","sequence":"additional","affiliation":[{"name":"Department of Translational Oncology, National Center for Tumor Diseases and German Cancer Research Center, 69120 Heidelberg, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christof","family":"von Kalle","sequence":"additional","affiliation":[{"name":"Department of Translational Oncology, National Center for Tumor Diseases and German Cancer Research Center, 69120 Heidelberg, Germany."},{"name":"German Cancer Consortium."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christoph","family":"Klein","sequence":"additional","affiliation":[{"name":"Dr. von Hauner Children\u2019s Hospital, Ludwig Maximilians University Munich, 80337 Munich, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"221","reference":[{"key":"e_1_3_2_2_2","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(94)90528-2"},{"key":"e_1_3_2_3_2","doi-asserted-by":"publisher","DOI":"10.1097\/MOH.0b013e3282f30448"},{"key":"e_1_3_2_4_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMp068209"},{"key":"e_1_3_2_5_2","doi-asserted-by":"publisher","DOI":"10.1038\/nri884"},{"key":"e_1_3_2_6_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.imbio.2009.06.009"},{"key":"e_1_3_2_7_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2008-12-115253"},{"key":"e_1_3_2_8_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2003-05-1480"},{"key":"e_1_3_2_9_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0022-3476(05)82002-5"},{"key":"e_1_3_2_10_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2010-11-319376"},{"key":"e_1_3_2_11_2","doi-asserted-by":"publisher","DOI":"10.1038\/nrg2971"},{"key":"e_1_3_2_12_2","doi-asserted-by":"publisher","DOI":"10.1038\/ni0610-457"},{"key":"e_1_3_2_13_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.288.5466.669"},{"key":"e_1_3_2_14_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa012616"},{"key":"e_1_3_2_15_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1000164"},{"key":"e_1_3_2_16_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa0805817"},{"key":"e_1_3_2_17_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.1070104"},{"key":"e_1_3_2_18_2","doi-asserted-by":"publisher","DOI":"10.1038\/nm1393"},{"key":"e_1_3_2_19_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.1091667"},{"key":"e_1_3_2_20_2","doi-asserted-by":"publisher","DOI":"10.1172\/JCI35700"},{"key":"e_1_3_2_21_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM200301163480314"},{"key":"e_1_3_2_22_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.1088547"},{"key":"e_1_3_2_23_2","doi-asserted-by":"publisher","DOI":"10.1172\/JCI35798"},{"key":"e_1_3_2_24_2","doi-asserted-by":"publisher","DOI":"10.1097\/MOH.0b013e3281900f01"},{"key":"e_1_3_2_25_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1003548"},{"key":"e_1_3_2_26_2","first-page":"390","article-title":"Development of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome.","volume":"8","author":"Boztug K.","year":"2006","unstructured":"Boztug K., Dewey R. A., Klein C., Development of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome. Curr. Opin. Mol. Ther. 8, 390\u2013395 (2006).","journal-title":"Curr. Opin. Mol. Ther."},{"key":"e_1_3_2_27_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2005-06-2336"},{"key":"e_1_3_2_28_2","doi-asserted-by":"publisher","DOI":"10.1084\/jem.20061893"},{"key":"e_1_3_2_29_2","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.162376099"},{"key":"e_1_3_2_30_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V86.10.3797.bloodjournal86103797"},{"key":"e_1_3_2_31_2","doi-asserted-by":"publisher","DOI":"10.1038\/nrc1299"},{"key":"e_1_3_2_32_2","doi-asserted-by":"publisher","DOI":"10.1038\/nm.2088"},{"key":"e_1_3_2_33_2","unstructured":"C. E. Shannon W. Weaver A Mathematical Theory of Communication (University of Illinois Press Urbana IL 1948)."},{"key":"e_1_3_2_34_2","doi-asserted-by":"publisher","DOI":"10.1038\/163688a0"},{"key":"e_1_3_2_35_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.leukres.2009.06.020"},{"key":"e_1_3_2_36_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2011-03-338707"},{"key":"e_1_3_2_37_2","doi-asserted-by":"publisher","DOI":"10.1038\/sj.leu.2404778"},{"key":"e_1_3_2_38_2","doi-asserted-by":"publisher","DOI":"10.1038\/leu.2010.224"},{"key":"e_1_3_2_39_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2004-12-4837"},{"key":"e_1_3_2_40_2","doi-asserted-by":"publisher","DOI":"10.1172\/JCI31661"},{"key":"e_1_3_2_41_2","doi-asserted-by":"publisher","DOI":"10.1172\/JCI31659"},{"key":"e_1_3_2_42_2","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2011.178"},{"key":"e_1_3_2_43_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.1233151"},{"key":"e_1_3_2_44_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.celrep.2013.09.040"},{"key":"e_1_3_2_45_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM200406103502422"},{"key":"e_1_3_2_46_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMra032207"},{"key":"e_1_3_2_47_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.exphem.2006.04.021"},{"key":"e_1_3_2_48_2","doi-asserted-by":"publisher","DOI":"10.1084\/jem.191.10.1699"},{"key":"e_1_3_2_49_2","doi-asserted-by":"publisher","DOI":"10.1111\/j.1399-0004.2008.01019.x"},{"key":"e_1_3_2_50_2","doi-asserted-by":"publisher","DOI":"10.1038\/sj.gt.3302863"},{"key":"e_1_3_2_51_2","doi-asserted-by":"publisher","DOI":"10.1016\/0167-5699(95)80117-0"},{"key":"e_1_3_2_52_2","unstructured":"C. Pannetier J. P. Levraud A. Lim J. Even P. Kourilsky The immunoscope approach for the analysis of T cell repertoires in The Antigen T Cell Receptor: Selected Protocols and Applications J. Oksenberg III Ed. (Landes Bioscience Austin TX 1997)."},{"key":"e_1_3_2_53_2","first-page":"1783","article-title":"Proposals for the immunological classification of acute leukemias. European Group for the Immunological Characterization of Leukemias (EGIL).","volume":"9","author":"Bene M. C.","year":"1995","unstructured":"Bene M. C., Castoldi G., Knapp W., Ludwig W. D., Matutes E., Orfao A., van\u2019t Veer M. B., Proposals for the immunological classification of acute leukemias. European Group for the Immunological Characterization of Leukemias (EGIL). Leukemia 9, 1783\u20131786 (1995).","journal-title":"Leukemia"},{"key":"e_1_3_2_54_2","doi-asserted-by":"crossref","unstructured":"B. Schlegelberger S. Metzke S. Harder R. Z\u00fchlke-Jenisch Y. Zhang R. Siebert Classical and Molecular Cytogenetics of Tumor Cells (Springer Berlin 1999) pp. 151\u2013185.","DOI":"10.1007\/978-3-642-59918-7_9"},{"key":"e_1_3_2_55_2","doi-asserted-by":"publisher","DOI":"10.1007\/s00439-009-0726-6"},{"key":"e_1_3_2_56_2","doi-asserted-by":"crossref","unstructured":"B. Schlegelberger S. Gesk Y. Zhang R. Siebert in FISH Technology (Springer Berlin 2002) pp. 442\u2013460.","DOI":"10.1007\/978-3-642-56404-8_34"},{"key":"e_1_3_2_57_2","doi-asserted-by":"publisher","DOI":"10.1038\/nprot.2010.87"},{"key":"e_1_3_2_58_2","doi-asserted-by":"publisher","DOI":"10.1038\/nmeth1103"},{"key":"e_1_3_2_59_2","doi-asserted-by":"publisher","DOI":"10.1101\/gr.229202"},{"key":"e_1_3_2_60_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0000570"}],"updated-by":[{"DOI":"10.1126\/scitranslmed.3010603","type":"correction","label":"Correction","source":"retraction-watch","updated":{"date-parts":[[2014,9,17]],"date-time":"2014-09-17T00:00:00Z","timestamp":1410912000000},"record-id":"8384"}],"container-title":["Science Translational Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.science.org\/doi\/pdf\/10.1126\/scitranslmed.3007280","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,1,16]],"date-time":"2024-01-16T12:53:21Z","timestamp":1705409601000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.science.org\/doi\/10.1126\/scitranslmed.3007280"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2014,3,12]]},"references-count":59,"journal-issue":{"issue":"227","published-print":{"date-parts":[[2014,3,12]]}},"alternative-id":["10.1126\/scitranslmed.3007280"],"URL":"https:\/\/doi.org\/10.1126\/scitranslmed.3007280","relation":{"has-review":[{"id-type":"doi","id":"10.3410\/f.718308660.793511924","asserted-by":"object"}]},"ISSN":["1946-6234","1946-6242"],"issn-type":[{"value":"1946-6234","type":"print"},{"value":"1946-6242","type":"electronic"}],"subject":[],"published":{"date-parts":[[2014,3,12]]}}}