{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,10]],"date-time":"2026-09-10T16:06:49Z","timestamp":1789056409022,"version":"build-2803163510"},"reference-count":25,"publisher":"Elsevier BV","issue":"3","license":[{"start":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T00:00:00Z","timestamp":1788220800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T00:00:00Z","timestamp":1788220800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2026,8,19]],"date-time":"2026-08-19T00:00:00Z","timestamp":1787097600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100009619","name":"Japan Agency for Medical Research and Development","doi-asserted-by":"publisher","award":["JP23mk0121273"],"award-info":[{"award-number":["JP23mk0121273"]}],"id":[{"id":"10.13039\/100009619","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100009619","name":"Japan Agency for Medical Research and Development","doi-asserted-by":"publisher","award":["JP22bm1223001"],"award-info":[{"award-number":["JP22bm1223001"]}],"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 Advances"],"published-print":{"date-parts":[[2026,9]]},"DOI":"10.1016\/j.omta.2026.201836","type":"journal-article","created":{"date-parts":[[2026,8,21]],"date-time":"2026-08-21T15:29:54Z","timestamp":1787326194000},"page":"201836","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":0,"title":["Single-cell vector copy number analysis of phenotypically defined long-term hematopoietic stem cells for gene therapy safety assessment"],"prefix":"10.1016","volume":"34","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8042-0069","authenticated-orcid":false,"given":"Yohei","family":"Sato","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Saki Kondo","family":"Matsushima","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yohta","family":"Shimada","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hiroshi","family":"Kobayashi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omta.2026.201836_bib1","doi-asserted-by":"crossref","first-page":"301","DOI":"10.1038\/nrg2985","article-title":"Ex vivo gene transfer and correction for cell-based therapies","volume":"12","author":"Naldini","year":"2011","journal-title":"Nat. Rev. Genet."},{"key":"10.1016\/j.omta.2026.201836_bib2","doi-asserted-by":"crossref","first-page":"351","DOI":"10.1038\/nature15818","article-title":"Gene therapy returns to centre stage","volume":"526","author":"Naldini","year":"2015","journal-title":"Nature"},{"key":"10.1016\/j.omta.2026.201836_bib3","doi-asserted-by":"crossref","first-page":"479","DOI":"10.1038\/gt.2010.163","article-title":"Quantification of lentiviral vector copy numbers in individual hematopoietic colony-forming cells shows vector dose-dependent effects on the frequency and level of transduction","volume":"18","author":"Charrier","year":"2011","journal-title":"Gene Ther."},{"key":"10.1016\/j.omta.2026.201836_bib4","doi-asserted-by":"crossref","first-page":"448","DOI":"10.1016\/j.omtm.2020.02.003","article-title":"Optimized Assessment of qPCR-Based Vector Copy Numbers as a Safety Parameter for GMP-Grade CAR T Cells and Monitoring of Frequency in Patients","volume":"17","author":"Kunz","year":"2020","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omta.2026.201836_bib5","doi-asserted-by":"crossref","first-page":"403","DOI":"10.1016\/j.omtm.2023.07.003","article-title":"Detection and quantification of integrated vector copy number by multiplex droplet digital PCR in dual-transduced CAR T cells","volume":"30","author":"Wang","year":"2023","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omta.2026.201836_bib6","doi-asserted-by":"crossref","first-page":"197","DOI":"10.1089\/hgtb.2016.059","article-title":"Application of Droplet Digital PCR for Estimating Vector Copy Number States in Stem Cell Gene Therapy","volume":"27","author":"Lin","year":"2016","journal-title":"Hum. Gene Ther. Methods"},{"key":"10.1016\/j.omta.2026.201836_bib7","doi-asserted-by":"crossref","first-page":"1954","DOI":"10.1016\/j.ymthe.2025.12.048","article-title":"Impact of vector copy number on critical quality attributes of retrovirally engineered T cells","volume":"34","author":"Norberg","year":"2026","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omta.2026.201836_bib8","doi-asserted-by":"crossref","first-page":"8","DOI":"10.1038\/s41588-023-01631-4","article-title":"Clonal dynamics of blood stem cells after gene therapy","volume":"56","author":"Danovi","year":"2024","journal-title":"Nat. Genet."},{"key":"10.1016\/j.omta.2026.201836_bib9","doi-asserted-by":"crossref","first-page":"819","DOI":"10.1111\/bjh.17175","article-title":"Clonal tracking of haematopoietic cells: insights and clinical implications","volume":"192","author":"Cordes","year":"2021","journal-title":"Br. J. Haematol."},{"key":"10.1016\/j.omta.2026.201836_bib10","doi-asserted-by":"crossref","first-page":"944","DOI":"10.1016\/j.omtm.2020.04.016","article-title":"Vector Copy Distribution at a Single-Cell Level Enhances Analytical Characterization of Gene-Modified Cell Therapies","volume":"17","author":"Santeramo","year":"2020","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omta.2026.201836_bib11","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.omta.2026.201836_bib12","doi-asserted-by":"crossref","first-page":"171","DOI":"10.1007\/978-1-0716-1771-7_12","article-title":"High-Throughput Multimodal Single-Cell Targeted DNA and Surface Protein Analysis Using the Mission Bio Tapestri Platform","volume":"2386","author":"Ruff","year":"2022","journal-title":"Methods Mol. Biol."},{"key":"10.1016\/j.omta.2026.201836_bib13","doi-asserted-by":"crossref","first-page":"509","DOI":"10.1182\/blood.2022018071","article-title":"Human CD34+ hematopoietic stem cell hierarchy: how far are we with its delineation at the most primitive level?","volume":"142","author":"Anjos-Afonso","year":"2023","journal-title":"Blood"},{"key":"10.1016\/j.omta.2026.201836_bib14","doi-asserted-by":"crossref","first-page":"162","DOI":"10.1038\/s41586-024-08250-x","article-title":"Long-term lineage commitment in haematopoietic stem cell gene therapy","volume":"636","author":"Calabria","year":"2024","journal-title":"Nature"},{"key":"10.1016\/j.omta.2026.201836_bib15","doi-asserted-by":"crossref","first-page":"2663","DOI":"10.1016\/j.ymthe.2025.04.029","article-title":"Clinical hematopoietic stem cell-based gene therapy","volume":"33","author":"John","year":"2025","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omta.2026.201836_bib16","doi-asserted-by":"crossref","first-page":"337","DOI":"10.1097\/MOH.0b013e3281900f01","article-title":"Insertional mutagenesis in gene therapy and stem cell biology","volume":"14","author":"Baum","year":"2007","journal-title":"Curr. Opin. Hematol."},{"key":"10.1016\/j.omta.2026.201836_bib17","article-title":"A Novel Branched DNA-Based Flowcytometric Method for Single-Cell Characterization of Gene Therapy Products and Expression of Therapeutic Genes","volume":"11","author":"Garcia-Perez","year":"2020","journal-title":"Front. Immunol."},{"key":"10.1016\/j.omta.2026.201836_bib19","doi-asserted-by":"crossref","first-page":"3175","DOI":"10.1038\/s41591-023-02636-6","article-title":"Clonal selection of hematopoietic stem cells after gene therapy for sickle cell disease","volume":"29","author":"Spencer Chapman","year":"2023","journal-title":"Nat. Med."},{"key":"10.1016\/j.omta.2026.201836_bib20","doi-asserted-by":"crossref","first-page":"5","DOI":"10.1016\/j.ymthe.2003.10.013","article-title":"Chance or necessity? Insertional mutagenesis in gene therapy and its consequences","volume":"9","author":"Baum","year":"2004","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omta.2026.201836_bib21","unstructured":"FDA CBER. OTAT Town Hall Series: Gene Therapy Chemistry, Manufacturing, and Controls. 2022."},{"key":"10.1016\/j.omta.2026.201836_bib22","unstructured":"European Medicines Agency. Guideline on the quality, non-clinical and clinical aspects of gene therapy medicinal products. EMA\/CAT\/80183\/2014. 2018."},{"key":"10.1016\/j.omta.2026.201836_bib23","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtm.2025.101472","article-title":"Interlaboratory assessment of candidate reference materials for lentiviral vector copy number and integration site measurements","volume":"33","author":"He","year":"2025","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omta.2026.201836_bib24","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtm.2023.101120","article-title":"Universal ddPCR-based assay for the determination of lentivirus infectious titer and lenti-modified cell vector copy number","volume":"31","author":"Kandell","year":"2023","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omta.2026.201836_bib25","doi-asserted-by":"crossref","DOI":"10.1038\/s41598-021-96045-9","article-title":"Comparison of seven single cell whole genome amplification commercial kits using targeted sequencing","volume":"11","author":"Biezuner","year":"2021","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omta.2026.201836_bib26","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtm.2025.101615","article-title":"Harnessing DNA barcoding to enhance and sustain polyclonality in gene-edited hematopoietic stem cells","volume":"33","author":"Ojeda-Perez","year":"2025","journal-title":"Mol. Ther. Methods Clin. Dev."}],"container-title":["Molecular Therapy Advances"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S3117387X26001710?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S3117387X26001710?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,9,10]],"date-time":"2026-09-10T15:13:57Z","timestamp":1789053237000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S3117387X26001710"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,9]]},"references-count":25,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2026,9]]}},"alternative-id":["S3117387X26001710"],"URL":"https:\/\/doi.org\/10.1016\/j.omta.2026.201836","relation":{},"ISSN":["3117-387X"],"issn-type":[{"value":"3117-387X","type":"print"}],"subject":[],"published":{"date-parts":[[2026,9]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Single-cell vector copy number analysis of phenotypically defined long-term hematopoietic stem cells for gene therapy safety assessment","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy Advances","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omta.2026.201836","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2026 The Author(s). Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}],"article-number":"201836"}}