{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,7]],"date-time":"2026-08-07T16:54:52Z","timestamp":1786121692547,"version":"build-2736575974"},"reference-count":66,"publisher":"Elsevier BV","issue":"8","license":[{"start":{"date-parts":[[2026,8,1]],"date-time":"2026-08-01T00:00:00Z","timestamp":1785542400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,8,1]],"date-time":"2026-08-01T00:00:00Z","timestamp":1785542400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2026,5,28]],"date-time":"2026-05-28T00:00:00Z","timestamp":1779926400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100018703","name":"European Innovation Council","doi-asserted-by":"publisher","award":["101070950"],"award-info":[{"award-number":["101070950"]}],"id":[{"id":"10.13039\/100018703","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000780","name":"European Commission","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000780","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100002426","name":"Fondazione Telethon","doi-asserted-by":"publisher","award":["SR-TIGET core grant 2016"],"award-info":[{"award-number":["SR-TIGET core grant 2016"]}],"id":[{"id":"10.13039\/501100002426","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100002426","name":"Fondazione Telethon","doi-asserted-by":"publisher","award":["SR-TIGET core grant 2021"],"award-info":[{"award-number":["SR-TIGET core grant 2021"]}],"id":[{"id":"10.13039\/501100002426","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100002426","name":"Fondazione Telethon","doi-asserted-by":"publisher","award":["C1\/3309"],"award-info":[{"award-number":["C1\/3309"]}],"id":[{"id":"10.13039\/501100002426","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003196","name":"Ministry of Health","doi-asserted-by":"publisher","award":["GR-2019-12369499"],"award-info":[{"award-number":["GR-2019-12369499"]}],"id":[{"id":"10.13039\/501100003196","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100031478","name":"NextGenerationEU","doi-asserted-by":"publisher","award":["G83C22000270001"],"award-info":[{"award-number":["G83C22000270001"]}],"id":[{"id":"10.13039\/100031478","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2026,8]]},"DOI":"10.1016\/j.ymthe.2026.05.014","type":"journal-article","created":{"date-parts":[[2026,5,26]],"date-time":"2026-05-26T16:20:05Z","timestamp":1779812405000},"page":"4581-4603","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":0,"title":["Ex vivo expansion of hematopoietic stem and progenitor cells from human mobilized peripheral blood for gene therapy applications"],"prefix":"10.1016","volume":"34","author":[{"given":"Erika","family":"Zonari","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9542-2884","authenticated-orcid":false,"given":"Matteo Maria","family":"Naldini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matteo","family":"Barcella","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Monica","family":"Volpin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesca","family":"Vincenti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giacomo","family":"Desantis","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Leila","family":"Hadadi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carolina","family":"Caserta","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ilaria","family":"Galasso","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Beatrice","family":"Martini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Federico","family":"Midena","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giacomo","family":"Farina","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roberta","family":"Vacca","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesca","family":"Tucci","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Leonardo","family":"Ormoli","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ilaria","family":"Visigalli","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michela","family":"Vezzoli","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dejan","family":"Lazarevic","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ivan","family":"Merelli","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephanie Z.","family":"Xie","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John E.","family":"Dick","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Raffaella","family":"Di Micco","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eugenio","family":"Montini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6024-4718","authenticated-orcid":false,"given":"Bernhard","family":"Gentner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymthe.2026.05.014_bib1","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.ymthe.2026.05.014_bib2","doi-asserted-by":"crossref","first-page":"926","DOI":"10.1038\/s41586-024-08128-y","article-title":"Clonal dynamics after allogeneic haematopoietic cell transplantation","volume":"635","author":"Spencer Chapman","year":"2024","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2026.05.014_bib3","doi-asserted-by":"crossref","first-page":"21","DOI":"10.1182\/blood.2023023788","article-title":"Bone marrow niches for hematopoietic stem cells: life span dynamics and adaptation to acute stress","volume":"144","author":"Hofmann","year":"2024","journal-title":"Blood"},{"key":"10.1016\/j.ymthe.2026.05.014_bib4","doi-asserted-by":"crossref","first-page":"2716","DOI":"10.3324\/haematol.2019.245688","article-title":"History of hematopoietic cell transplantation: challenges and progress","volume":"105","author":"Granot","year":"2020","journal-title":"haematol."},{"key":"10.1016\/j.ymthe.2026.05.014_bib5","doi-asserted-by":"crossref","first-page":"232","DOI":"10.1038\/nm.2080","article-title":"Notch-mediated expansion of human cord blood progenitor cells capable of rapid myeloid reconstitution","volume":"16","author":"Delaney","year":"2010","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2026.05.014_bib6","doi-asserted-by":"crossref","first-page":"1429","DOI":"10.1182\/blood.2021011719","article-title":"Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase 3 randomized study","volume":"138","author":"Horwitz","year":"2021","journal-title":"Blood"},{"key":"10.1016\/j.ymthe.2026.05.014_bib7","doi-asserted-by":"crossref","first-page":"e134","DOI":"10.1016\/S2352-3026(19)30202-9","article-title":"Hematopoietic stem cell transplantation using single UM171-expanded cord blood: a single-arm, phase 1-2 safety and feasibility study","volume":"7","author":"Cohen","year":"2020","journal-title":"Lancet Haematol."},{"key":"10.1016\/j.ymthe.2026.05.014_bib8","doi-asserted-by":"crossref","first-page":"144","DOI":"10.1016\/j.stem.2015.10.004","article-title":"Phase I\/II Trial of StemRegenin-1 Expanded Umbilical Cord Blood Hematopoietic Stem Cells Supports Testing as a Stand-Alone Graft","volume":"18","author":"Wagner","year":"2016","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.ymthe.2026.05.014_bib9","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1038\/nature13035","article-title":"Haematopoietic stem cells require a highly regulated protein synthesis rate","volume":"509","author":"Signer","year":"2014","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2026.05.014_bib10","doi-asserted-by":"crossref","first-page":"1778","DOI":"10.1038\/nm.2984","article-title":"Maintenance of hematopoietic stem cells through regulation of Wnt and mTOR pathways","volume":"18","author":"Huang","year":"2012","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2026.05.014_bib11","doi-asserted-by":"crossref","first-page":"729","DOI":"10.1182\/blood.2023021426","article-title":"Adaptation to ex vivo culture reduces human hematopoietic stem cell activity independently of the cell cycle","volume":"144","author":"Johnson","year":"2024","journal-title":"Blood"},{"key":"10.1016\/j.ymthe.2026.05.014_bib12","doi-asserted-by":"crossref","first-page":"1509","DOI":"10.1126\/science.1256337","article-title":"Pyrimidoindole derivatives are agonists of human hematopoietic stem cell self-renewal","volume":"345","author":"Fares","year":"2014","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2026.05.014_bib13","doi-asserted-by":"crossref","first-page":"48","DOI":"10.1016\/j.stem.2020.12.002","article-title":"UM171 Preserves Epigenetic Marks that Are Reduced in Ex Vivo Culture of Human HSCs via Potentiation of the CLR3-KBTBD4 Complex","volume":"28","author":"Chagraoui","year":"2021","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.ymthe.2026.05.014_bib14","doi-asserted-by":"crossref","first-page":"2378","DOI":"10.1172\/JCI70313","article-title":"Epigenetic reprogramming induces the expansion of cord blood stem cells","volume":"124","author":"Chaurasia","year":"2014","journal-title":"J. Clin. Invest."},{"key":"10.1016\/j.ymthe.2026.05.014_bib15","doi-asserted-by":"crossref","first-page":"2410","DOI":"10.1182\/blood.2020005357","article-title":"The BET inhibitor CPI203 promotes ex vivo expansion of cord blood long-term repopulating HSCs and megakaryocytes","volume":"136","author":"Hua","year":"2020","journal-title":"Blood"},{"key":"10.1016\/j.ymthe.2026.05.014_bib16","doi-asserted-by":"crossref","first-page":"3121","DOI":"10.1172\/JCI74556","article-title":"Umbilical cord blood expansion with nicotinamide provides long-term multilineage engraftment","volume":"124","author":"Horwitz","year":"2014","journal-title":"J. Clin. Invest."},{"key":"10.1016\/j.ymthe.2026.05.014_bib17","doi-asserted-by":"crossref","first-page":"508","DOI":"10.1038\/nature17665","article-title":"Musashi-2 attenuates AHR signalling to expand human haematopoietic stem cells","volume":"532","author":"Rentas","year":"2016","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2026.05.014_bib18","doi-asserted-by":"crossref","first-page":"1345","DOI":"10.1126\/science.1191536","article-title":"Aryl hydrocarbon receptor antagonists promote the expansion of human hematopoietic stem cells","volume":"329","author":"Boitano","year":"2010","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2026.05.014_bib19","doi-asserted-by":"crossref","first-page":"360","DOI":"10.1038\/nm.4477","article-title":"Antagonism of PPAR-\u03b3 signaling expands human hematopoietic stem and progenitor cells by enhancing glycolysis","volume":"24","author":"Guo","year":"2018","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2026.05.014_bib20","doi-asserted-by":"crossref","first-page":"405","DOI":"10.1016\/j.stem.2019.02.012","article-title":"The NAD-Booster Nicotinamide Riboside Potently Stimulates Hematopoiesis through Increased Mitochondrial Clearance","volume":"24","author":"Vannini","year":"2019","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.ymthe.2026.05.014_bib21","doi-asserted-by":"crossref","first-page":"639","DOI":"10.1016\/j.stem.2019.09.008","article-title":"Sphingolipid Modulation Activates Proteostasis Programs to Govern Human Hematopoietic Stem Cell Self-Renewal","volume":"25","author":"Xie","year":"2019","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.ymthe.2026.05.014_bib22","doi-asserted-by":"crossref","first-page":"378","DOI":"10.1038\/s41590-025-02087-w","article-title":"Transcriptional activation of regenerative hematopoiesis via microenvironmental sensing","volume":"26","author":"Itkin","year":"2025","journal-title":"Nat. Immunol."},{"key":"10.1016\/j.ymthe.2026.05.014_bib23","doi-asserted-by":"crossref","first-page":"127","DOI":"10.1038\/s41586-023-05739-9","article-title":"Chemically defined cytokine-free expansion of human haematopoietic stem cells","volume":"615","author":"Sakurai","year":"2023","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2026.05.014_bib24","doi-asserted-by":"crossref","first-page":"1566","DOI":"10.1038\/s41591-019-0601-5","article-title":"Expansion of primitive human hematopoietic stem cells by culture in a zwitterionic hydrogel","volume":"25","author":"Bai","year":"2019","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2026.05.014_bib25","doi-asserted-by":"crossref","DOI":"10.3389\/fbioe.2022.968086","article-title":"Functionalized 3D scaffolds for engineering the hematopoietic niche","volume":"10","author":"Bruschi","year":"2022","journal-title":"Front. Bioeng. Biotechnol."},{"key":"10.1016\/j.ymthe.2026.05.014_bib26","doi-asserted-by":"crossref","first-page":"129","DOI":"10.1016\/j.actbio.2021.03.061","article-title":"Rebuilding the hematopoietic stem cell niche: Recent developments and future prospects","volume":"132","author":"Chatterjee","year":"2021","journal-title":"Acta Biomater."},{"key":"10.1016\/j.ymthe.2026.05.014_bib27","doi-asserted-by":"crossref","first-page":"216","DOI":"10.1038\/s41576-020-00298-5","article-title":"Gene therapy using haematopoietic stem and progenitor cells","volume":"22","author":"Ferrari","year":"2021","journal-title":"Nat. Rev. Genet."},{"key":"10.1016\/j.ymthe.2026.05.014_bib28","doi-asserted-by":"crossref","first-page":"549","DOI":"10.1016\/j.stem.2023.04.014","article-title":"Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy","volume":"30","author":"Ferrari","year":"2023","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.ymthe.2026.05.014_bib29","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-023-38448-y","article-title":"Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy","volume":"14","author":"Scala","year":"2023","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2026.05.014_bib30","doi-asserted-by":"crossref","first-page":"1929","DOI":"10.1056\/NEJMoa2106596","article-title":"Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome","volume":"385","author":"Gentner","year":"2021","journal-title":"N. Engl. J. Med. Overseas. Ed."},{"key":"10.1016\/j.ymthe.2026.05.014_bib31","doi-asserted-by":"crossref","first-page":"977","DOI":"10.1016\/j.stemcr.2017.02.010","article-title":"Efficient Ex Vivo Engineering and Expansion of Highly Purified Human Hematopoietic Stem and Progenitor Cell Populations for Gene Therapy","volume":"8","author":"Zonari","year":"2017","journal-title":"Stem Cell Rep."},{"key":"10.1016\/j.ymthe.2026.05.014_bib32","doi-asserted-by":"crossref","first-page":"531","DOI":"10.1002\/sctm.19-0199","article-title":"Expansion and preservation of the functional activity of adult hematopoietic stem cells cultured ex vivo with a histone deacetylase inhibitor","volume":"9","author":"Zimran","year":"2020","journal-title":"Stem Cells Transl. Med."},{"key":"10.1016\/j.ymthe.2026.05.014_bib33","doi-asserted-by":"crossref","first-page":"1198","DOI":"10.15252\/emmm.201707922","article-title":"Lentiviral vectors escape innate sensing but trigger p53 in human hematopoietic stem and progenitor cells","volume":"9","author":"Piras","year":"2017","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.ymthe.2026.05.014_bib34","doi-asserted-by":"crossref","first-page":"74","DOI":"10.3324\/haematol.2019.238261","article-title":"Expanded circulating hematopoietic stem\/progenitor cells as novel cell source for the treatment of TCIRG1 osteopetrosis","volume":"106","author":"Capo","year":"2020","journal-title":"haematol."},{"key":"10.1016\/j.ymthe.2026.05.014_bib35","doi-asserted-by":"crossref","first-page":"3919","DOI":"10.1182\/blood.V89.11.3919","article-title":"Primitive human hematopoietic cells are enriched in cord blood compared with adult bone marrow or mobilized peripheral blood as measured by the quantitative in vivo SCID-repopulating cell assay","volume":"89","author":"Wang","year":"1997","journal-title":"Blood"},{"key":"10.1016\/j.ymthe.2026.05.014_bib36","doi-asserted-by":"crossref","first-page":"1990","DOI":"10.1182\/blood.2022017174","article-title":"Aging alters the cell cycle control and mitogenic signaling responses of human hematopoietic stem cells","volume":"141","author":"Hammond","year":"2023","journal-title":"Blood"},{"key":"10.1016\/j.ymthe.2026.05.014_bib37","doi-asserted-by":"crossref","first-page":"942","DOI":"10.1038\/s41409-022-01662-1","article-title":"CD14 positive cells accelerate hematopoietic stem cell engraftment","volume":"57","author":"Pievani","year":"2022","journal-title":"Bone Marrow Transpl."},{"key":"10.1016\/j.ymthe.2026.05.014_bib38","doi-asserted-by":"crossref","first-page":"1937","DOI":"10.1182\/blood.2023022666","article-title":"Circulating hematopoietic stem\/progenitor cell subsets contribute to human hematopoietic homeostasis","volume":"143","author":"Quaranta","year":"2024","journal-title":"Blood"},{"key":"10.1016\/j.ymthe.2026.05.014_bib39","doi-asserted-by":"crossref","first-page":"423","DOI":"10.1016\/j.cell.2017.11.018","article-title":"Intrinsic Immunity Shapes Viral Resistance of Stem Cells","volume":"172","author":"Wu","year":"2018","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2026.05.014_bib40","article-title":"A p38 MAPK-ROS axis fuels proliferation stress and DNA damage during CRISPR-Cas9 gene editing in hematopoietic stem and progenitor cells","volume":"5","author":"della Volpe","year":"2024","journal-title":"Cell Rep. Med."},{"key":"10.1016\/j.ymthe.2026.05.014_bib41","doi-asserted-by":"crossref","first-page":"218","DOI":"10.1126\/science.1201219","article-title":"Isolation of single human hematopoietic stem cells capable of long-term multilineage engraftment","volume":"333","author":"Notta","year":"2011","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2026.05.014_bib42","doi-asserted-by":"crossref","first-page":"2048","DOI":"10.1038\/s41467-022-29675-w","article-title":"Single cell analyses identify a highly regenerative and homogenous human CD34+ hematopoietic stem cell population","volume":"13","author":"Anjos-Afonso","year":"2022","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2026.05.014_bib43","doi-asserted-by":"crossref","first-page":"195","DOI":"10.1038\/s41467-024-55225-7","article-title":"Deciphering the effect of UM171 on human hematopoietic progenitor cell fate through clonal analysis","volume":"16","author":"Coulombe","year":"2025","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2026.05.014_bib44","doi-asserted-by":"crossref","first-page":"723","DOI":"10.1038\/s41590-021-00925-1","article-title":"A latent subset of human hematopoietic stem cells resists regenerative stress to preserve stemness","volume":"22","author":"Kaufmann","year":"2021","journal-title":"Nat. Immunol."},{"key":"10.1016\/j.ymthe.2026.05.014_bib45","doi-asserted-by":"crossref","first-page":"799","DOI":"10.1016\/j.stem.2012.09.001","article-title":"Attenuation of miR-126 activity expands HSC in vivo without exhaustion","volume":"11","author":"Lechman","year":"2012","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.ymthe.2026.05.014_bib46","doi-asserted-by":"crossref","first-page":"302","DOI":"10.1016\/j.stem.2015.01.017","article-title":"CDK6 levels regulate quiescence exit in human hematopoietic stem cells","volume":"16","author":"Laurenti","year":"2015","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.ymthe.2026.05.014_bib47","article-title":"Reducing oxidative stress improves ex vivo polymer-based human haematopoietic stem and progenitor cell culture and gene editing","author":"Bozhilov","year":"2024","journal-title":"bioRxiv"},{"key":"10.1016\/j.ymthe.2026.05.014_bib48","doi-asserted-by":"crossref","first-page":"86","DOI":"10.1007\/s40778-020-00172-4","article-title":"Mechanoregulation in hematopoiesis and hematologic disorders","volume":"6","author":"Horton","year":"2020","journal-title":"Curr. Stem Cell Rep."},{"key":"10.1016\/j.ymthe.2026.05.014_bib49","doi-asserted-by":"crossref","DOI":"10.1016\/j.bone.2022.116519","article-title":"Osteoclast rich osteopetrosis due to defects in the TCIRG1 gene","volume":"165","author":"Capo","year":"2022","journal-title":"Bone"},{"key":"10.1016\/j.ymthe.2026.05.014_bib50","doi-asserted-by":"crossref","DOI":"10.1126\/science.1233158","article-title":"Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy","volume":"341","author":"Biffi","year":"2013","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2026.05.014_bib51","doi-asserted-by":"crossref","DOI":"10.1126\/science.1233151","article-title":"Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome","volume":"341","author":"Aiuti","year":"2013","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2026.05.014_bib52","doi-asserted-by":"crossref","first-page":"217","DOI":"10.1038\/76095","article-title":"Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences","volume":"25","author":"Follenzi","year":"2000","journal-title":"Nat. Genet."},{"key":"10.1016\/j.ymthe.2026.05.014_bib53","doi-asserted-by":"crossref","first-page":"411","DOI":"10.1016\/j.omtm.2020.10.009","article-title":"Laboratory-Scale Lentiviral Vector Production and Purification for Enhanced Ex Vivo and In Vivo Genetic Engineering","volume":"19","author":"Soldi","year":"2020","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2026.05.014_bib54","article-title":"Senescence and inflammation are unintended adverse consequences of CRISPR-Cas9\/AAV6-mediated gene editing in hematopoietic stem cells","volume":"6","author":"Conti","year":"2025","journal-title":"Cell Rep. Med."},{"key":"10.1016\/j.ymthe.2026.05.014_bib55","doi-asserted-by":"crossref","first-page":"3558","DOI":"10.1016\/j.molcel.2023.09.006","article-title":"A fluorophore-conjugated reagent enabling rapid detection, isolation and live tracking of senescent cells","volume":"83","author":"Magkouta","year":"2023","journal-title":"Mol. Cell"},{"key":"10.1016\/j.ymthe.2026.05.014_bib56","doi-asserted-by":"crossref","first-page":"1458","DOI":"10.1038\/s41591-021-01389-4","article-title":"Retrieval of vector integration sites from cell-free DNA","volume":"27","author":"Cesana","year":"2021","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2026.05.014_bib57","doi-asserted-by":"crossref","first-page":"520","DOI":"10.1186\/s12859-017-1937-9","article-title":"VISPA2: a scalable pipeline for high-throughput identification and annotation of vector integration sites","volume":"18","author":"Spinozzi","year":"2017","journal-title":"BMC Bioinformatics"},{"key":"10.1016\/j.ymthe.2026.05.014_bib58","doi-asserted-by":"crossref","DOI":"10.1093\/bib\/bbac551","article-title":"ISAnalytics enables longitudinal and high-throughput clonal tracking studies in hematopoietic stem cell gene therapy applications","volume":"24","author":"Pais","year":"2023","journal-title":"Brief. Bioinform."},{"key":"10.1016\/j.ymthe.2026.05.014_bib59","doi-asserted-by":"crossref","first-page":"755","DOI":"10.1093\/bioinformatics\/bts004","article-title":"Estimating abundances of retroviral insertion sites from DNA fragment length data","volume":"28","author":"Berry","year":"2012","journal-title":"Bioinformatics"},{"key":"10.1016\/j.ymthe.2026.05.014_bib60","doi-asserted-by":"crossref","first-page":"5332","DOI":"10.1182\/blood-2010-09-306761","article-title":"Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection","volume":"117","author":"Biffi","year":"2011","journal-title":"Blood"},{"key":"10.1016\/j.ymthe.2026.05.014_bib61","doi-asserted-by":"crossref","first-page":"2991","DOI":"10.1038\/s41596-021-00529-x","article-title":"BAR-Seq clonal tracking of gene-edited cells","volume":"16","author":"Ferrari","year":"2021","journal-title":"Nat. Protoc."},{"key":"10.1016\/j.ymthe.2026.05.014_bib62","doi-asserted-by":"crossref","DOI":"10.1016\/j.xinn.2021.100141","article-title":"clusterProfiler 4.0: A universal enrichment tool for interpreting omics data","volume":"2","author":"Wu","year":"2021","journal-title":"The Innovation"},{"key":"10.1016\/j.ymthe.2026.05.014_bib63","doi-asserted-by":"crossref","first-page":"3573","DOI":"10.1016\/j.cell.2021.04.048","article-title":"Integrated analysis of multimodal single-cell data","volume":"184","author":"Hao","year":"2021","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2026.05.014_bib64","doi-asserted-by":"crossref","first-page":"163","DOI":"10.1038\/s41590-018-0276-y","article-title":"Reference-based analysis of lung single-cell sequencing reveals a transitional profibrotic macrophage","volume":"20","author":"Aran","year":"2019","journal-title":"Nat. Immunol."},{"key":"10.1016\/j.ymthe.2026.05.014_bib65","doi-asserted-by":"crossref","first-page":"2642","DOI":"10.1093\/bioinformatics\/btac141","article-title":"scGate: marker-based purification of cell types from heterogeneous single-cell RNA-seq datasets","volume":"38","author":"Andreatta","year":"2022","journal-title":"Bioinformatics"},{"key":"10.1016\/j.ymthe.2026.05.014_bib66","doi-asserted-by":"crossref","first-page":"70","DOI":"10.1016\/j.jim.2009.06.008","article-title":"ELDA: extreme limiting dilution analysis for comparing depleted and enriched populations in stem cell and other assays","volume":"347","author":"Hu","year":"2009","journal-title":"J. Immunol. Methods"}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001626004016?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001626004016?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,8,7]],"date-time":"2026-08-07T06:39:19Z","timestamp":1786084759000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001626004016"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,8]]},"references-count":66,"journal-issue":{"issue":"8","published-print":{"date-parts":[[2026,8]]}},"alternative-id":["S1525001626004016"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2026.05.014","relation":{"has-preprint":[{"id-type":"doi","id":"10.64898\/2026.04.08.716064","asserted-by":"object"}]},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2026,8]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Ex vivo expansion of hematopoietic stem and progenitor cells from human mobilized peripheral blood for gene therapy applications","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2026.05.014","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2026 The Authors. Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}]}}