{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,7]],"date-time":"2026-10-07T02:45:58Z","timestamp":1791341158764,"version":"4.3.1"},"reference-count":22,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2006,1,6]],"date-time":"2006-01-06T00:00:00Z","timestamp":1136505600000},"content-version":"vor","delay-in-days":66,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Annals of the New York Academy of Sciences"],"published-print":{"date-parts":[[2005,11]]},"abstract":"<jats:p><jats:bold>A<jats:sc>bstract<\/jats:sc>: <\/jats:bold> Recent success in the long\u2010term correction of mouse models of human \u03b2\u2010thalassemia and sickle cell anemia by lentiviral vectors and evidence of high gene transfer and expression in transduced human hematopoietic cells have led to a first clinical trial of gene therapy for the disease. A LentiGlobin vector containing a \u03b2\u2010globin gene (\u03b2<jats:sup>A\u2010T87Q<\/jats:sup>) that produces a hemoglobin (Hb\u03b2<jats:sup>A\u2010T87Q<\/jats:sup>) that can be distinguished from normal hemoglobin will be used. The LentiGlobin vector is self\u2010inactivating and contains large elements of the \u03b2\u2010globin locus control region as well as chromatin insulators and other features that should prevent untoward events. The study will be done in Paris with Eliane Gluckman as the principal investigator and Philippe Leboulch as scientific director.<\/jats:p>","DOI":"10.1196\/annals.1345.007","type":"journal-article","created":{"date-parts":[[2005,12,8]],"date-time":"2005-12-08T23:18:51Z","timestamp":1134083931000},"page":"308-316","source":"Crossref","is-referenced-by-count":143,"title":["A Phase I\/II Clinical Trial of \u03b2\u2010Globin Gene Therapy for \u03b2\u2010Thalassemia"],"prefix":"10.1111","volume":"1054","author":[{"given":"ARTHUR","family":"BANK","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"RONALD","family":"DORAZIO","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"PHILIPPE","family":"LEBOULCH","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2009,7,8]]},"reference":[{"key":"e_1_2_4_2_2","doi-asserted-by":"publisher","DOI":"10.1002\/bies.950181210"},{"key":"e_1_2_4_3_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.288.5466.669"},{"key":"e_1_2_4_4_2","doi-asserted-by":"publisher","DOI":"10.1002\/j.1460-2075.1994.tb06605.x"},{"key":"e_1_2_4_5_2","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.92.15.6728"},{"key":"e_1_2_4_6_2","first-page":"S106","article-title":"Successful incorporation of extended LCR and \u03b2 globin gene constructs in oncoretroviral and lentiviral vectors","volume":"1","author":"Alkan O.","year":"2000","journal-title":"Mol. Ther."},{"key":"e_1_2_4_7_2","doi-asserted-by":"publisher","DOI":"10.1038\/35017565"},{"key":"e_1_2_4_8_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.1065806"},{"key":"e_1_2_4_9_2","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.212507099"},{"key":"e_1_2_4_10_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2002-10-3305"},{"key":"e_1_2_4_11_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2003-03-0677"},{"key":"e_1_2_4_12_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2004-04-1427"},{"key":"e_1_2_4_13_2","doi-asserted-by":"publisher","DOI":"10.1172\/JCI200421838"},{"key":"e_1_2_4_14_2","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.1998.16.1.165"},{"key":"e_1_2_4_15_2","doi-asserted-by":"publisher","DOI":"10.1038\/76225"},{"key":"e_1_2_4_16_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V92.7.2269"},{"key":"e_1_2_4_17_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V96.3.902.015k40_902_909"},{"key":"e_1_2_4_18_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V97.6.1888"},{"key":"e_1_2_4_19_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM200301163480314"},{"key":"e_1_2_4_20_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.1088547"},{"key":"e_1_2_4_21_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM200406103502422"},{"key":"e_1_2_4_22_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pmed.0020010"},{"key":"e_1_2_4_23_2","doi-asserted-by":"publisher","DOI":"10.1016\/S1525-0016(03)00212-0"}],"container-title":["Annals of the New York Academy of Sciences"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1196%2Fannals.1345.007","content-type":"unspecified","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/nyaspubs.onlinelibrary.wiley.com\/doi\/pdf\/10.1196\/annals.1345.007","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,25]],"date-time":"2023-09-25T20:54:16Z","timestamp":1695675256000},"score":1,"resource":{"primary":{"URL":"https:\/\/nyaspubs.onlinelibrary.wiley.com\/doi\/10.1196\/annals.1345.007"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2005,11]]},"references-count":22,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2005,11]]}},"alternative-id":["10.1196\/annals.1345.007"],"URL":"https:\/\/doi.org\/10.1196\/annals.1345.007","archive":["Portico"],"relation":{},"ISSN":["0077-8923","1749-6632"],"issn-type":[{"value":"0077-8923","type":"print"},{"value":"1749-6632","type":"electronic"}],"subject":[],"published":{"date-parts":[[2005,11]]}}}