{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,16]],"date-time":"2026-09-16T06:08:35Z","timestamp":1789538915711,"version":"build-2803163510"},"reference-count":21,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2015,2,6]],"date-time":"2015-02-06T00:00:00Z","timestamp":1423180800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"name":"DHHS","award":["U01CA151837"],"award-info":[{"award-number":["U01CA151837"]}]},{"name":"DHHS","award":["R01CA154471"],"award-info":[{"award-number":["R01CA154471"]}]},{"name":"DHHS","award":["DP2OD007246"],"award-info":[{"award-number":["DP2OD007246"]}]},{"name":"DHHS","award":["P30CA014089"],"award-info":[{"award-number":["P30CA014089"]}]},{"name":"Nautica Triathalon","award":["Leukemia Research Funds"],"award-info":[{"award-number":["Leukemia Research Funds"]}]},{"name":"USC Keck School of Medicine","award":["RMI Award"],"award-info":[{"award-number":["RMI Award"]}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Br J Haematol"],"published-print":{"date-parts":[[2015,5]]},"abstract":"Summary<\/jats:title>B\u2010precursor acute lymphoblastic leukaemia (BPL<\/jats:styled-content>) is the most common form of cancer in children and adolescents. Our recent studies have demonstrated that CD<\/jats:styled-content>22<\/jats:italic>\u0394E12 is a characteristic genetic defect of therapy\u2010refractory clones in paediatric BPL<\/jats:styled-content> and implicated the CD<\/jats:styled-content>22<\/jats:italic>\u0394E12 genetic defect in the aggressive biology of relapsed or therapy\u2010refractory paediatric BPL<\/jats:styled-content>. The purpose of the present study is to evaluate the biological significance of the CD<\/jats:styled-content>22<\/jats:italic>\u0394E12 molecular lesion in BPL<\/jats:styled-content> and determine if it could serve as a molecular target for RNA<\/jats:styled-content> interference (RNA<\/jats:styled-content>i) therapy. Here we report a previously unrecognized causal link between CD<\/jats:styled-content>22<\/jats:italic>\u0394E12 and aggressive biology of human BPL<\/jats:styled-content> cells by demonstrating that siRNA<\/jats:styled-content>\u2010mediated knockdown of CD<\/jats:styled-content>22<\/jats:italic>\u0394E12 in primary leukaemic B\u2010cell precursors is associated with a marked inhibition of their clonogenicity. Additionally, we report a nanoscale liposomal formulation of CD<\/jats:styled-content>22<\/jats:italic>\u0394E12\u2010specific siRNA<\/jats:styled-content> with potent in vitro<\/jats:italic> and in vivo<\/jats:italic> anti\u2010leukaemic activity against primary human BPL<\/jats:styled-content> cells as a first\u2010in\u2010class RNA<\/jats:styled-content>i therapeutic candidate targeting CD<\/jats:styled-content>22<\/jats:italic>\u0394E12.<\/jats:p>","DOI":"10.1111\/bjh.13306","type":"journal-article","created":{"date-parts":[[2015,2,6]],"date-time":"2015-02-06T23:47:50Z","timestamp":1423266470000},"page":"401-414","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["CD<\/scp>22<\/i>\u0394E12 as a molecular target for RNA<\/scp>i therapy"],"prefix":"10.1111","volume":"169","author":[{"given":"Fatih M.","family":"Uckun","sequence":"first","affiliation":[{"name":"Children's Center for Cancer and Blood Diseases Children's Hospital Los Angeles (CHLA) Los Angeles CA USA"},{"name":"Division of Hematology\u2010Oncology Department of Pediatrics University of Southern California Keck School of Medicine (USC KSOM) Los Angeles CA USA"},{"name":"Translational and Clinical Sciences Program Norris Comprehensive Cancer Center University of Southern California Keck School of Medicine (USC KSOM) Los Angeles CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hong","family":"Ma","sequence":"additional","affiliation":[{"name":"Children's Center for Cancer and Blood Diseases Children's Hospital Los Angeles (CHLA) Los Angeles CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jianjun","family":"Cheng","sequence":"additional","affiliation":[{"name":"Department of Materials Science and Engineering University of Illinois at Urbana\u2010Champaign Urbana IL USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dorothea 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