{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,22]],"date-time":"2026-10-22T07:29:47Z","timestamp":1792654187664,"version":"4.5.0"},"reference-count":36,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2019,12,1]],"date-time":"2019-12-01T00:00:00Z","timestamp":1575158400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2019,12,1]],"date-time":"2019-12-01T00:00:00Z","timestamp":1575158400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2019,11,7]],"date-time":"2019-11-07T00:00:00Z","timestamp":1573084800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000062","name":"National Institute of Diabetes and Digestive and Kidney Diseases","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000062","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000050","name":"National Heart, Lung, and Blood Institute","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000050","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy - Methods & Clinical Development"],"published-print":{"date-parts":[[2019,12]]},"DOI":"10.1016\/j.omtm.2019.10.017","type":"journal-article","created":{"date-parts":[[2019,11,11]],"date-time":"2019-11-11T19:17:29Z","timestamp":1573499849000},"page":"430-437","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":30,"special_numbering":"C","title":["Low-Dose Busulfan Reduces Human CD34+ Cell Doses Required for Engraftment in c-kit Mutant Immunodeficient Mice"],"prefix":"10.1016","volume":"15","author":[{"given":"Alexis","family":"Leonard","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Morgan","family":"Yapundich","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tina","family":"Nassehi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jackson","family":"Gamer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claire M.","family":"Drysdale","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Juan J.","family":"Haro-Mora","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Selami","family":"Demirci","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthew M.","family":"Hsieh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Naoya","family":"Uchida","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John F.","family":"Tisdale","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2019.10.017_bib1","doi-asserted-by":"crossref","first-page":"193","DOI":"10.1182\/blood-2010-02-271841","article-title":"Comparison of human cord blood engraftment between immunocompromised mouse strains","volume":"116","author":"McDermott","year":"2010","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2019.10.017_bib2","doi-asserted-by":"crossref","first-page":"6477","DOI":"10.4049\/jimmunol.174.10.6477","article-title":"Human lymphoid and myeloid cell development in NOD\/LtSz-scid IL2R gamma null mice engrafted with mobilized human hemopoietic stem cells","volume":"174","author":"Shultz","year":"2005","journal-title":"J.\u00a0Immunol."},{"key":"10.1016\/j.omtm.2019.10.017_bib3","doi-asserted-by":"crossref","first-page":"717","DOI":"10.1182\/blood-2011-06-361626","article-title":"Ionizing radiation-induced expression of INK4a\/ARF in murine bone marrow-derived stromal cell populations interferes with bone marrow homeostasis","volume":"119","author":"Carbonneau","year":"2012","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2019.10.017_bib4","doi-asserted-by":"crossref","first-page":"4013","DOI":"10.1182\/blood.V89.11.4013","article-title":"Potential and distribution of transplanted hematopoietic stem cells in a nonablated mouse model","volume":"89","author":"Nilsson","year":"1997","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2019.10.017_bib5","doi-asserted-by":"crossref","first-page":"3105","DOI":"10.1182\/blood-2013-07-515619","article-title":"Total body irradiation causes long-term mouse BM injury via induction of HSC premature senescence in an Ink4a- and Arf-independent manner","volume":"123","author":"Shao","year":"2014","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2019.10.017_bib6","doi-asserted-by":"crossref","first-page":"3629","DOI":"10.1182\/blood-2011-08-373621","article-title":"An acute negative bystander effect of \u03b3-irradiated recipients on transplanted hematopoietic stem cells","volume":"119","author":"Shen","year":"2012","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2019.10.017_bib7","doi-asserted-by":"crossref","first-page":"1415","DOI":"10.1016\/j.exphem.2007.05.009","article-title":"Low-dose parenteral busulfan provides an extended window for the infusion of hematopoietic stem cells in murine hosts","volume":"35","author":"Hsieh","year":"2007","journal-title":"Exp. Hematol."},{"key":"10.1016\/j.omtm.2019.10.017_bib8","doi-asserted-by":"crossref","first-page":"1335","DOI":"10.1038\/bmt.2013.60","article-title":"Sirolimus and post transplant Cy synergistically maintain mixed chimerism in a mismatched murine model","volume":"48","author":"Fitzhugh","year":"2013","journal-title":"Bone Marrow Transplant."},{"key":"10.1016\/j.omtm.2019.10.017_bib9","doi-asserted-by":"crossref","first-page":"1465","DOI":"10.3727\/096368910X314161","article-title":"The assessment of human erythroid output in NOD\/SCID mice reconstituted with human hematopoietic stem cells","volume":"19","author":"Hayakawa","year":"2010","journal-title":"Cell Transplant."},{"key":"10.1016\/j.omtm.2019.10.017_bib10","doi-asserted-by":"crossref","first-page":"175","DOI":"10.1634\/stemcells.2008-0583","article-title":"Busulfan produces efficient human cell engraftment in NOD\/LtSz-Scid IL2Rgamma(null) mice","volume":"27","author":"Hayakawa","year":"2009","journal-title":"Stem Cells"},{"key":"10.1016\/j.omtm.2019.10.017_bib11","doi-asserted-by":"crossref","first-page":"171","DOI":"10.1016\/j.stemcr.2014.12.005","article-title":"Nonirradiated NOD,B6.SCID Il2r\u03b3-\/- Kit(W41\/W41) (NBSGW) mice support multilineage engraftment of human hematopoietic cells","volume":"4","author":"McIntosh","year":"2015","journal-title":"Stem Cell Reports"},{"key":"10.1016\/j.omtm.2019.10.017_bib12","doi-asserted-by":"crossref","first-page":"591","DOI":"10.1016\/j.stemcr.2016.08.005","article-title":"Improved Human Erythropoiesis and Platelet Formation in Humanized NSGW41 Mice","volume":"7","author":"Rahmig","year":"2016","journal-title":"Stem Cell Reports"},{"key":"10.1016\/j.omtm.2019.10.017_bib13","doi-asserted-by":"crossref","first-page":"E513","DOI":"10.1002\/ajh.24805","article-title":"Developmentally-faithful and effective human erythropoiesis in immunodeficient and Kit mutant mice","volume":"92","author":"Fiorini","year":"2017","journal-title":"Am. J. Hematol."},{"key":"10.1016\/j.omtm.2019.10.017_bib14","doi-asserted-by":"crossref","first-page":"227","DOI":"10.1016\/j.stem.2014.06.001","article-title":"Kit regulates HSC engraftment across the human-mouse species barrier","volume":"15","author":"Cosgun","year":"2014","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.omtm.2019.10.017_bib15","doi-asserted-by":"crossref","first-page":"425","DOI":"10.1016\/j.stemcr.2016.07.002","article-title":"Enhanced Reconstitution of Human Erythropoiesis and Thrombopoiesis in an Immunodeficient Mouse Model with Kit(Wv) Mutations","volume":"7","author":"Yurino","year":"2016","journal-title":"Stem Cell Reports"},{"key":"10.1016\/j.omtm.2019.10.017_bib16","doi-asserted-by":"crossref","first-page":"84","DOI":"10.1016\/j.clim.2009.12.008","article-title":"Parameters for establishing humanized mouse models to study human immunity: analysis of human hematopoietic stem cell engraftment in three immunodeficient strains of mice bearing the IL2rgamma(null) mutation","volume":"135","author":"Brehm","year":"2010","journal-title":"Clin. Immunol."},{"key":"10.1016\/j.omtm.2019.10.017_bib17","doi-asserted-by":"crossref","first-page":"748","DOI":"10.1016\/j.it.2018.07.001","article-title":"Humanized Mice for the Study of Immuno-Oncology","volume":"39","author":"De La Rochere","year":"2018","journal-title":"Trends Immunol."},{"key":"10.1016\/j.omtm.2019.10.017_bib18","doi-asserted-by":"crossref","first-page":"1389","DOI":"10.1016\/j.ymthe.2019.05.014","article-title":"Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates","volume":"27","author":"Romero","year":"2019","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2019.10.017_bib19","doi-asserted-by":"crossref","first-page":"167","DOI":"10.1016\/j.omtm.2018.10.014","article-title":"Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling \u03b2AS3 Globin for Gene Therapy for Sickle Cell Disease","volume":"11","author":"Poletti","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2019.10.017_bib20","doi-asserted-by":"crossref","first-page":"358","DOI":"10.1038\/nprot.2017.143","article-title":"CRISPR\/Cas9 genome editing in human hematopoietic stem cells","volume":"13","author":"Bak","year":"2018","journal-title":"Nat. Protoc."},{"key":"10.1016\/j.omtm.2019.10.017_bib21","doi-asserted-by":"crossref","first-page":"15045","DOI":"10.1038\/mtm.2015.45","article-title":"Amelioration of murine sickle cell disease by nonablative conditioning and \u03b3-globin gene-corrected bone marrow cells","volume":"2","author":"Pestina","year":"2015","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2019.10.017_bib22","doi-asserted-by":"crossref","first-page":"137","DOI":"10.1016\/j.omtm.2016.12.009","article-title":"Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34+ Hematopoietic Stem and Progenitor Cells","volume":"4","author":"Chang","year":"2017","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2019.10.017_bib23","doi-asserted-by":"crossref","first-page":"2597","DOI":"10.1182\/blood-2014-12-615948","article-title":"Correction of the sickle cell disease mutation in human hematopoietic stem\/progenitor cells","volume":"125","author":"Hoban","year":"2015","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2019.10.017_bib24","first-page":"67930","article-title":"\u03b2-Globin gene transfer to human bone marrow for sickle cell disease","author":"Romero","year":"2013","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2019.10.017_bib25","doi-asserted-by":"crossref","first-page":"1153","DOI":"10.1089\/hum.2018.061","article-title":"Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study","volume":"29","author":"Urbinati","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2019.10.017_bib26","doi-asserted-by":"crossref","first-page":"71","DOI":"10.1089\/scd.2016.0230","article-title":"Gene Therapy with Hematopoietic Stem Cells: The Diseased Bone Marrow\u2019s Point of View","volume":"26","author":"Cavazzana","year":"2017","journal-title":"Stem Cells Dev."},{"key":"10.1016\/j.omtm.2019.10.017_bib27","doi-asserted-by":"crossref","first-page":"286","DOI":"10.1111\/bjh.15902","article-title":"Bone marrow characterization in sickle cell disease: inflammation and stress erythropoiesis lead to suboptimal CD34 recovery","volume":"186","author":"Leonard","year":"2019","journal-title":"Br. J. Haematol."},{"key":"10.1016\/j.omtm.2019.10.017_bib28","doi-asserted-by":"crossref","first-page":"1926","DOI":"10.1158\/1078-0432.CCR-07-5134","article-title":"Regulation of hematopoietic stem cells by the steel factor\/KIT signaling pathway","volume":"14","author":"Kent","year":"2008","journal-title":"Clin. Cancer Res."},{"key":"10.1016\/j.omtm.2019.10.017_bib29","doi-asserted-by":"crossref","first-page":"267","DOI":"10.1038\/nmeth.1309","article-title":"Hematopoietic stem cell transplantation without irradiation","volume":"6","author":"Waskow","year":"2009","journal-title":"Nat. Methods"},{"key":"10.1016\/j.omtm.2019.10.017_bib30","doi-asserted-by":"crossref","first-page":"1296","DOI":"10.1126\/science.1149726","article-title":"Efficient transplantation via antibody-based clearance of hematopoietic stem cell niches","volume":"318","author":"Czechowicz","year":"2007","journal-title":"Science"},{"key":"10.1016\/j.omtm.2019.10.017_bib31","doi-asserted-by":"crossref","first-page":"2045","DOI":"10.4049\/jimmunol.180.4.2045","article-title":"Kit regulates maintenance of quiescent hematopoietic stem cells","volume":"180","author":"Thor\u00e9n","year":"2008","journal-title":"J.\u00a0Immunol."},{"key":"10.1016\/j.omtm.2019.10.017_bib32","doi-asserted-by":"crossref","first-page":"9854","DOI":"10.1128\/JVI.00357-09","article-title":"Development of a human immunodeficiency virus type 1-based lentiviral vector that allows efficient transduction of both human and rhesus blood cells","volume":"83","author":"Uchida","year":"2009","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.omtm.2019.10.017_bib33","doi-asserted-by":"crossref","first-page":"1078","DOI":"10.1038\/gt.2011.63","article-title":"Optimal conditions for lentiviral transduction of engrafting human CD34+ cells","volume":"18","author":"Uchida","year":"2011","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2019.10.017_bib34","doi-asserted-by":"crossref","first-page":"187","DOI":"10.1016\/j.omtm.2019.01.005","article-title":"High-Efficiency Lentiviral Transduction of Human CD34+ Cells in High-Density Culture with Poloxamer and Prostaglandin E2","volume":"13","author":"Uchida","year":"2019","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2019.10.017_bib35","doi-asserted-by":"crossref","first-page":"e122","DOI":"10.1038\/mtna.2013.49","article-title":"Integration-specific In\u00a0Vitro Evaluation of Lentivirally Transduced Rhesus CD34(+) Cells Correlates With In\u00a0Vivo Vector Copy Number","volume":"2","author":"Uchida","year":"2013","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtm.2019.10.017_bib36","doi-asserted-by":"crossref","first-page":"456","DOI":"10.1007\/BF02986613","article-title":"Generation of a chimeric mouse reconstituted with green fluorescent protein-positive bone marrow cells: a useful model for studying the behavior of bone marrow cells in regeneration in\u00a0vivo","volume":"77","author":"Hayakawa","year":"2003","journal-title":"Int. J. Hematol."}],"container-title":["Molecular Therapy - Methods & Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050119301251?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050119301251?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,6]],"date-time":"2025-11-06T09:24:04Z","timestamp":1762421044000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050119301251"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,12]]},"references-count":36,"alternative-id":["S2329050119301251"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2019.10.017","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2019,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Low-Dose Busulfan Reduces Human CD34+ Cell Doses Required for Engraftment in c-kit Mutant Immunodeficient Mice","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2019.10.017","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"name":"copyright","label":"Copyright"}]}}