{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,15]],"date-time":"2026-05-15T18:17:54Z","timestamp":1778869074412,"version":"3.51.4"},"reference-count":31,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2021,12,1]],"date-time":"2021-12-01T00:00:00Z","timestamp":1638316800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2021,12,1]],"date-time":"2021-12-01T00:00:00Z","timestamp":1638316800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2021,10,13]],"date-time":"2021-10-13T00:00:00Z","timestamp":1634083200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100007786","name":"National Center for Child Health and Development","doi-asserted-by":"publisher","award":["28-1"],"award-info":[{"award-number":["28-1"]}],"id":[{"id":"10.13039\/100007786","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100007786","name":"National Center for Child Health and Development","doi-asserted-by":"publisher","award":["2020B-2"],"award-info":[{"award-number":["2020B-2"]}],"id":[{"id":"10.13039\/100007786","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100009619","name":"Japan Agency for Medical Research and Development","doi-asserted-by":"publisher","award":["17ek0109167h0002"],"award-info":[{"award-number":["17ek0109167h0002"]}],"id":[{"id":"10.13039\/100009619","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":[[2021,12]]},"DOI":"10.1016\/j.omtm.2021.10.003","type":"journal-article","created":{"date-parts":[[2021,10,19]],"date-time":"2021-10-19T07:59:16Z","timestamp":1634630356000},"page":"424-433","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":7,"special_numbering":"C","title":["Nonconditioned ADA-SCID gene therapy reveals ADA requirement in the hematopoietic system and clonal dominance of vector-marked clones"],"prefix":"10.1016","volume":"23","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-9610-0900","authenticated-orcid":false,"given":"Toru","family":"Uchiyama","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sirirat","family":"Takahashi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kazuhiko","family":"Nakabayashi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kohji","family":"Okamura","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kaori","family":"Edasawa","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6347-3521","authenticated-orcid":false,"given":"Masafumi","family":"Yamada","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nobuyuki","family":"Watanabe","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Emi","family":"Mochizuki","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Toru","family":"Yasuda","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Akane","family":"Miura","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5145-1774","authenticated-orcid":false,"given":"Motohiro","family":"Kato","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1520-7007","authenticated-orcid":false,"given":"Daisuke","family":"Tomizawa","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Makoto","family":"Otsu","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tadashi","family":"Ariga","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9449-2066","authenticated-orcid":false,"given":"Masafumi","family":"Onodera","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2021.10.003_bib1","first-page":"291","article-title":"Adenosine deaminase deficiency: clinical expression, molecular basis, and therapy","volume":"35","author":"Hershfield","year":"1998","journal-title":"Semin. Hematol."},{"key":"10.1016\/j.omtm.2021.10.003_bib2","doi-asserted-by":"crossref","first-page":"3524","DOI":"10.1182\/blood-2009-06-189209","article-title":"How I treat ADA deficiency","volume":"114","author":"Gaspar","year":"2009","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2021.10.003_bib3","doi-asserted-by":"crossref","first-page":"174","DOI":"10.1016\/j.gene.2013.03.098","article-title":"Gene therapy for PIDs: progress, pitfalls and prospects","volume":"525","author":"Mukherjee","year":"2013","journal-title":"Gene"},{"key":"10.1016\/j.omtm.2021.10.003_bib4","doi-asserted-by":"crossref","first-page":"789","DOI":"10.1517\/14712598.2014.895811","article-title":"Gene therapy for inherited immunodeficiency","volume":"14","author":"Touzot","year":"2014","journal-title":"Expert Opin. Biol. Ther."},{"key":"10.1016\/j.omtm.2021.10.003_bib5","doi-asserted-by":"crossref","first-page":"2410","DOI":"10.1126\/science.1070104","article-title":"Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning","volume":"296","author":"Aiuti","year":"2002","journal-title":"Science"},{"key":"10.1016\/j.omtm.2021.10.003_bib6","doi-asserted-by":"crossref","first-page":"505","DOI":"10.1016\/j.ymthe.2006.06.007","article-title":"Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning","volume":"14","author":"Gaspar","year":"2006","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.10.003_bib7","doi-asserted-by":"crossref","first-page":"3635","DOI":"10.1182\/blood-2012-02-400937","article-title":"Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans","volume":"120","author":"Candotti","year":"2012","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2021.10.003_bib8","doi-asserted-by":"crossref","first-page":"97ra80","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":"10.1016\/j.omtm.2021.10.003_bib9","doi-asserted-by":"crossref","first-page":"2624","DOI":"10.1182\/blood-2016-12-756734","article-title":"Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients","volume":"129","author":"Cooper","year":"2017","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2021.10.003_bib10","doi-asserted-by":"crossref","first-page":"1689","DOI":"10.1172\/JCI90367","article-title":"Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency","volume":"127","author":"Shaw","year":"2017","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2021.10.003_bib11","doi-asserted-by":"crossref","first-page":"384","DOI":"10.1007\/s10875-015-0157-1","article-title":"Outcomes in two Japanese adenosine deaminase-deficiency patients treated by stem cell gene therapy with no cytoreductive conditioning","volume":"35","author":"Otsu","year":"2015","journal-title":"J.\u00a0Clin. Immunol."},{"key":"10.1016\/j.omtm.2021.10.003_bib12","doi-asserted-by":"crossref","first-page":"8","DOI":"10.1016\/j.omtm.2017.05.005","article-title":"Single Cell-Based Vector Tracing in Patients with ADA-SCID Treated with Stem Cell Gene Therapy","volume":"6","author":"Igarashi","year":"2017","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2021.10.003_bib13","doi-asserted-by":"crossref","first-page":"30","DOI":"10.1182\/blood.V91.1.30","article-title":"Successful peripheral T-lymphocyte-directed gene transfer for a patient with severe combined immune deficiency caused by adenosine deaminase deficiency","volume":"91","author":"Onodera","year":"1998","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2021.10.003_bib14","doi-asserted-by":"crossref","first-page":"89","DOI":"10.1002\/emmm.201000108","article-title":"Integration profile of retroviral vector in gene therapy treated patients is cell-specific according to gene expression and chromatin conformation of target cell","volume":"3","author":"Biasco","year":"2011","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.omtm.2021.10.003_bib15","first-page":"235","article-title":"New insights into TRAP1 pathway","volume":"2","author":"Matassa","year":"2012","journal-title":"Am. J. Cancer Res."},{"key":"10.1016\/j.omtm.2021.10.003_bib16","doi-asserted-by":"crossref","first-page":"58","DOI":"10.3389\/fonc.2017.00058","article-title":"The Chaperone TRAP1 As a Modulator of the Mitochondrial Adaptations in Cancer Cells","volume":"7","author":"Masgras","year":"2017","journal-title":"Front. Oncol."},{"key":"10.1016\/j.omtm.2021.10.003_bib17","doi-asserted-by":"crossref","first-page":"836","DOI":"10.1038\/nature02041","article-title":"Identification of the haematopoietic stem cell niche and control of the niche size","volume":"425","author":"Zhang","year":"2003","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2021.10.003_bib18","doi-asserted-by":"crossref","first-page":"841","DOI":"10.1038\/nature02040","article-title":"Osteoblastic cells regulate the haematopoietic stem cell niche","volume":"425","author":"Calvi","year":"2003","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2021.10.003_bib19","doi-asserted-by":"crossref","first-page":"537","DOI":"10.1084\/jem.20110994","article-title":"Osteoclasts promote the formation of hematopoietic stem cell niches in the bone marrow","volume":"209","author":"Mansour","year":"2012","journal-title":"J.\u00a0Exp. Med."},{"key":"10.1016\/j.omtm.2021.10.003_bib20","doi-asserted-by":"crossref","first-page":"3216","DOI":"10.1182\/blood-2009-03-209221","article-title":"ADA-deficient SCID is associated with a specific microenvironment and bone phenotype characterized by RANKL\/OPG imbalance and osteoblast insufficiency","volume":"114","author":"Sauer","year":"2009","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2021.10.003_bib21","first-page":"152","article-title":"Role of adenosine deaminase in the early stages of precursor T\u00a0cell maturation","volume":"44","author":"Shore","year":"1981","journal-title":"Clin. Exp. Immunol."},{"key":"10.1016\/j.omtm.2021.10.003_bib22","doi-asserted-by":"crossref","first-page":"5011","DOI":"10.1073\/pnas.75.10.5011","article-title":"Purinogenic immunodeficiency diseases: selective toxicity of deoxyribonucleosides for T\u00a0cells","volume":"75","author":"Mitchell","year":"1978","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2021.10.003_bib23","doi-asserted-by":"crossref","first-page":"1475","DOI":"10.1172\/JCI109606","article-title":"Purinogenic immunodeficiency diseases. Differential effects of deoxyadenosine and deoxyguanosine on DNA synthesis in human T lymphoblasts","volume":"64","author":"Wilson","year":"1979","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2021.10.003_bib24","doi-asserted-by":"crossref","first-page":"395","DOI":"10.1172\/JCI0215683","article-title":"Adenosine kinase inhibition promotes survival of fetal adenosine deaminase-deficient thymocytes by blocking dATP accumulation","volume":"110","author":"Van De Wiele","year":"2002","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2021.10.003_bib25","doi-asserted-by":"crossref","first-page":"1600","DOI":"10.1182\/blood.V90.4.1600","article-title":"Role of A2a extracellular adenosine receptor-mediated signaling in adenosine-mediated inhibition of T-cell activation and expansion","volume":"90","author":"Huang","year":"1997","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2021.10.003_bib26","doi-asserted-by":"crossref","first-page":"1528","DOI":"10.4049\/jimmunol.168.4.1528","article-title":"Cutting edge: CD4 and CD8 T\u00a0cells are intrinsically different in their proliferative responses","volume":"168","author":"Foulds","year":"2002","journal-title":"J.\u00a0Immunol."},{"key":"10.1016\/j.omtm.2021.10.003_bib27","doi-asserted-by":"crossref","first-page":"40","DOI":"10.1016\/j.cellimm.2008.01.003","article-title":"Quantitative analysis of T\u00a0cell homeostatic proliferation","volume":"250","author":"Li","year":"2007","journal-title":"Cell. Immunol."},{"key":"10.1016\/j.omtm.2021.10.003_bib28","doi-asserted-by":"crossref","first-page":"127","DOI":"10.1046\/j.1365-2567.2002.01447.x","article-title":"Qualitative differences between na\u00efve and memory T\u00a0cells","volume":"106","author":"Berard","year":"2002","journal-title":"Immunology"},{"key":"10.1016\/j.omtm.2021.10.003_bib29","doi-asserted-by":"crossref","first-page":"e1005417","DOI":"10.1371\/journal.pcbi.1005417","article-title":"Modelling T\u00a0cell proliferation: Dynamics heterogeneity depending on cell differentiation, age, and genetic background","volume":"13","author":"Vibert","year":"2017","journal-title":"PLoS Comput. Biol."},{"key":"10.1016\/j.omtm.2021.10.003_bib30","doi-asserted-by":"crossref","first-page":"799","DOI":"10.1016\/j.jaci.2013.12.1043","article-title":"B-cell development and functions and therapeutic options in adenosine deaminase-deficient patients","volume":"133","author":"Brigida","year":"2014","journal-title":"J.\u00a0Allergy Clin. Immunol."},{"key":"10.1016\/j.omtm.2021.10.003_bib31","doi-asserted-by":"crossref","first-page":"2688","DOI":"10.1182\/blood-2011-01-329359","article-title":"Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency","volume":"118","author":"Sokolic","year":"2011","journal-title":"Blood"}],"container-title":["Molecular Therapy - Methods & Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050121001601?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050121001601?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,5,15]],"date-time":"2026-05-15T17:53:59Z","timestamp":1778867639000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050121001601"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,12]]},"references-count":31,"alternative-id":["S2329050121001601"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2021.10.003","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2021,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Nonconditioned ADA-SCID gene therapy reveals ADA requirement in the hematopoietic system and clonal dominance of vector-marked clones","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2021.10.003","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2021 The Authors.","name":"copyright","label":"Copyright"}]}}