{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,10]],"date-time":"2026-07-10T15:41:13Z","timestamp":1783698073034,"version":"3.55.0"},"reference-count":40,"publisher":"Elsevier BV","issue":"6","license":[{"start":{"date-parts":[[2024,6,1]],"date-time":"2024-06-01T00:00:00Z","timestamp":1717200000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2024,6,1]],"date-time":"2024-06-01T00:00:00Z","timestamp":1717200000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,6,5]],"date-time":"2025-06-05T00:00:00Z","timestamp":1749081600000},"content-version":"vor","delay-in-days":369,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2024,6]]},"DOI":"10.1016\/j.ymthe.2024.04.001","type":"journal-article","created":{"date-parts":[[2024,4,6]],"date-time":"2024-04-06T09:01:42Z","timestamp":1712394102000},"page":"1643-1657","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":5,"title":["TALEN-mediated intron editing of HSPCs enables transgene expression restricted to the myeloid lineage"],"prefix":"10.1016","volume":"32","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-5147-9144","authenticated-orcid":false,"given":"Eduardo","family":"Seclen","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessica C.","family":"Jang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aminah O.","family":"Lawal","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sylvain","family":"Pulicani","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alex","family":"Boyne","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diane","family":"Tkach","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexandre","family":"Juillerat","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Philippe","family":"Duchateau","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julien","family":"Valton","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymthe.2024.04.001_bib1","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":"2020","journal-title":"Nat. Rev. Genet."},{"key":"10.1016\/j.ymthe.2024.04.001_bib2","doi-asserted-by":"crossref","first-page":"83","DOI":"10.1016\/j.omtm.2019.11.013","article-title":"Non-toxic HSC Transplantation-Based Macrophage\/Microglia-Mediated GDNF Delivery for Parkinson\u2019s Disease","volume":"17","author":"Chen","year":"2020","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2024.04.001_bib3","doi-asserted-by":"crossref","first-page":"1630","DOI":"10.1056\/NEJMoa1700554","article-title":"Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy","volume":"377","author":"Eichler","year":"2017","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.ymthe.2024.04.001_bib4","doi-asserted-by":"crossref","first-page":"1479","DOI":"10.1056\/NEJMoa1705342","article-title":"Gene Therapy in Patients with Transfusion-Dependent \u03b2-Thalassemia","volume":"378","author":"Thompson","year":"2018","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.ymthe.2024.04.001_bib5","doi-asserted-by":"crossref","first-page":"E953","DOI":"10.1097\/HS9.0000000000000953","article-title":"Current and Future Perspective in Hematopoietic Stem Progenitor Cell-gene Therapy for Inborn Errors of Metabolism","volume":"7","author":"Tucci","year":"2023","journal-title":"HemaSphere"},{"key":"10.1016\/j.ymthe.2024.04.001_bib6","article-title":"Elivaldogene autotemcel approved for treatment of cerebral adrenoleukodystrophy (CALD) in males: A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG)","volume":"1","author":"Scott","year":"2023","journal-title":"Genet. Med. Open"},{"key":"10.1016\/j.ymthe.2024.04.001_bib7","doi-asserted-by":"crossref","first-page":"814","DOI":"10.1089\/hum.2018.169","article-title":"New Human Chromosomal Sites with \u201cSafe Harbor\u201d Potential for Targeted Transgene Insertion. Hum","volume":"30","author":"Pellenz","year":"2019","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2024.04.001_bib8","doi-asserted-by":"crossref","DOI":"10.3390\/pharmaceutics15051329","article-title":"Homology-Directed-Repair-Based Genome Editing in HSPCs for the Treatment of Inborn Errors of Immunity and Blood Disorders","volume":"15","author":"Allen","year":"2023","journal-title":"Pharmaceutics"},{"key":"10.1016\/j.ymthe.2024.04.001_bib9","doi-asserted-by":"crossref","first-page":"718","DOI":"10.1038\/mt.2008.5","article-title":"Physiological promoters reduce the genotoxic risk of integrating gene vectors","volume":"16","author":"Zychlinski","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2024.04.001_bib10","doi-asserted-by":"crossref","first-page":"1857","DOI":"10.1182\/blood-2009-12-256461","article-title":"The galactocerebrosidase enzyme contributes to the maintenance of a functional hematopoietic stem cell niche","volume":"116","author":"Visigalli","year":"2010","journal-title":"Blood"},{"key":"10.1016\/j.ymthe.2024.04.001_bib11","article-title":"Engineering monocyte\/macrophage-specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing","volume":"11","author":"Scharenberg","year":"2020","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2024.04.001_bib12","doi-asserted-by":"crossref","first-page":"1123","DOI":"10.1080\/14712598.2018.1536119","article-title":"Promise of gene therapy to treat sickle cell disease","volume":"18","author":"Romero","year":"2018","journal-title":"Expert Opin. Biol. Ther."},{"key":"10.1016\/j.ymthe.2024.04.001_bib13","doi-asserted-by":"crossref","first-page":"eadh8162","DOI":"10.1126\/scitranslmed.adh8162","article-title":"Exonic knockout and\u00a0knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency","volume":"16","author":"Castiello","year":"2024","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.ymthe.2024.04.001_bib14","first-page":"530","article-title":"Myeloid Engraftment in Humanized Mice: Impact of Granulocyte-Colony Stimulating Factor Treatment and Transgenic Mouse","volume":"25","author":"Coughlan","year":"2016","journal-title":"Strain"},{"key":"10.1016\/j.ymthe.2024.04.001_bib15","doi-asserted-by":"crossref","first-page":"3076","DOI":"10.1182\/blood-2010-08-301507","article-title":"Development of human CD4+FoxP3+ regulatory T\u00a0cells in human stem cell factor\u2013, granulocyte-macrophage colony-stimulating factor\u2013, and interleukin-3\u2013expressing NOD-SCID IL2R\u03b3null humanized mice","volume":"117","author":"Billerbeck","year":"2011","journal-title":"Blood"},{"key":"10.1016\/j.ymthe.2024.04.001_bib16","article-title":"Gene Correction for SCID-X1 in Long-Term Hematopoietic Stem Cells","volume":"3","author":"Pavel-Dinu","year":"2018","journal-title":"bioRxiv"},{"key":"10.1016\/j.ymthe.2024.04.001_bib17","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-019-11962-8","article-title":"Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I","volume":"10","author":"Gomez-Ospina","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2024.04.001_bib18","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.ymthe.2024.04.001_bib19","doi-asserted-by":"crossref","first-page":"3209","DOI":"10.1016\/j.ymthe.2022.05.022","article-title":"High-throughput analysis of hematopoietic stem cell engraftment after intravenous and intracerebroventricular dosing","volume":"30","author":"Plasschaert","year":"2022","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2024.04.001_bib20","doi-asserted-by":"crossref","first-page":"392","DOI":"10.1016\/j.omtm.2022.04.009","article-title":"Improved engraftment and therapeutic efficacy by human genome-edited hematopoietic stem cells with Busulfan-based myeloablation","volume":"25","author":"Poletto","year":"2022","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2024.04.001_bib21","doi-asserted-by":"crossref","first-page":"198","DOI":"10.3389\/fncel.2020.00198","article-title":"Overview of General and Discriminating Markers of Differential Microglia Phenotypes","volume":"14","author":"Jurga","year":"2020","journal-title":"Front. Cell. Neurosci."},{"key":"10.1016\/j.ymthe.2024.04.001_bib22","doi-asserted-by":"crossref","first-page":"195","DOI":"10.1007\/s12026-009-8101-1","article-title":"Leukocyte integrins and their ligand interactions","volume":"45","author":"Hyun","year":"2009","journal-title":"Immunol. Res."},{"key":"10.1016\/j.ymthe.2024.04.001_bib23","doi-asserted-by":"crossref","first-page":"1822","DOI":"10.1007\/s12015-022-10331-1","article-title":"Improved and Flexible HDR Editing by Targeting Introns in iPSCs","volume":"18","author":"Fu","year":"2022","journal-title":"Stem Cell Rev. Rep."},{"key":"10.1016\/j.ymthe.2024.04.001_bib24","doi-asserted-by":"crossref","first-page":"424","DOI":"10.1016\/j.cj.2019.03.006","article-title":"Intron-targeted gene insertion in rice using CRISPR\/Cas9: A case study of the Pi-ta gene","volume":"36","author":"Xu","year":"2020","journal-title":"Crop J."},{"key":"10.1016\/j.ymthe.2024.04.001_bib25","doi-asserted-by":"crossref","first-page":"111","DOI":"10.1007\/s00018-022-04152-1","article-title":"Efficient gene editing through an intronic selection marker in cells","volume":"79","author":"Wang","year":"2022","journal-title":"Cell. Mol. Life Sci."},{"key":"10.1016\/j.ymthe.2024.04.001_bib26","series-title":"Knock-in Tagging in Zebrafish Facilitated by Insertion into Non-coding Regions","author":"Levic","year":"2021"},{"key":"10.1016\/j.ymthe.2024.04.001_bib27","doi-asserted-by":"crossref","first-page":"634","DOI":"10.1038\/cr.2015.43","article-title":"Intron targeting-mediated and endogenous gene integrity-maintaining knockin in zebrafish using the CRISPR\/Cas9 system","volume":"25","author":"Li","year":"2015","journal-title":"Cell Res."},{"key":"10.1016\/j.ymthe.2024.04.001_bib28","doi-asserted-by":"crossref","first-page":"765","DOI":"10.1038\/nbt.4192","article-title":"Repair of double-strand breaks induced by CRISPR\u2013Cas9 leads to large deletions and complex rearrangements","author":"Kosicki","year":"2018","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2024.04.001_bib29","doi-asserted-by":"crossref","first-page":"178","DOI":"10.1016\/j.ymthe.2018.10.018","article-title":"ZFN-Mediated In\u00a0Vivo Genome Editing Corrects Murine Hurler Syndrome","volume":"27","author":"Ou","year":"2019","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2024.04.001_bib30","doi-asserted-by":"crossref","first-page":"493","DOI":"10.1016\/j.immuni.2009.01.014","article-title":"Genome-wide lineage-specific transcriptional networks underscore Ikaros-dependent lymphoid priming in Hematopoietic Stem Cells","volume":"30","author":"Ng","year":"2009","journal-title":"Immunity"},{"key":"10.1016\/j.ymthe.2024.04.001_bib31","article-title":"Rapamycin Promotes the Expansion of Myeloid Cells by Increasing G-CSF Expression in Mesenchymal Stem Cells","volume":"10","author":"Li","year":"2022","journal-title":"Front. Cell Dev. Biol."},{"key":"10.1016\/j.ymthe.2024.04.001_bib32","doi-asserted-by":"crossref","first-page":"1709","DOI":"10.1016\/B978-0-444-59565-2.00040-X","article-title":"Gaucher disease","volume":"113","author":"Mignot","year":"2013","journal-title":"Handb. Clin. Neurol."},{"key":"10.1016\/j.ymthe.2024.04.001_bib33","doi-asserted-by":"crossref","DOI":"10.3389\/fncel.2020.00198","article-title":"Overview of General and Discriminating Markers of Differential Microglia Phenotypes","volume":"14","author":"Jurga","year":"2020","journal-title":"Front. Cell. Neurosci."},{"key":"10.1016\/j.ymthe.2024.04.001_bib34","article-title":"Genetically engineered microglia-like cells have therapeutic potential for neurodegenerative disease","volume":"3","author":"Plasschaert","year":"2021","journal-title":"bioRxiv"},{"key":"10.1016\/j.ymthe.2024.04.001_bib35","doi-asserted-by":"crossref","first-page":"S71","DOI":"10.1016\/j.bbi.2010.12.018","article-title":"Targeting the hematopoietic system for the treatment of Alzheimer\u2019s disease","volume":"25","author":"Lampron","year":"2011","journal-title":"Brain Behav. Immun."},{"key":"10.1016\/j.ymthe.2024.04.001_bib36","doi-asserted-by":"crossref","DOI":"10.1016\/j.celrep.2020.108093","article-title":"Controlled Cycling and Quiescence Enables Efficient HDR in Engraftment-Enriched Adult Hematopoietic Stem and Progenitor Cells","volume":"32","author":"Shin","year":"2020","journal-title":"Cell Rep."},{"key":"10.1016\/j.ymthe.2024.04.001_bib37","doi-asserted-by":"crossref","first-page":"551","DOI":"10.1016\/j.stem.2019.02.019","article-title":"Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response","volume":"24","author":"Schiroli","year":"2019","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.ymthe.2024.04.001_bib38","doi-asserted-by":"crossref","first-page":"277","DOI":"10.1016\/j.omtn.2019.05.025","article-title":"In\u00a0Vivo Outcome of Homology-Directed Repair at the HBB Gene in HSC Using Alternative Donor Template Delivery Methods","volume":"17","author":"Pattabhi","year":"2019","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.ymthe.2024.04.001_bib39","doi-asserted-by":"crossref","first-page":"4350","DOI":"10.1182\/blood.V124.21.4350.4350","article-title":"Transient Bcl-XL Overexpression in Donor Stem Cells Increases Efficacy of Hematopoietic Stem Cell Transplantation without Increasing the Risk of Leukemogenesis","volume":"124","author":"Erlacher","year":"2014","journal-title":"Blood"},{"key":"10.1016\/j.ymthe.2024.04.001_bib40","doi-asserted-by":"crossref","first-page":"95","DOI":"10.1038\/nbt.4021","article-title":"Inhibition of 53BP1 favors homology-dependent DNA repair and increases CRISPR-Cas9 genome-editing efficiency","volume":"36","author":"Canny","year":"2018","journal-title":"Nat. Biotechnol."}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S152500162400217X?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S152500162400217X?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,4,21]],"date-time":"2026-04-21T20:22:26Z","timestamp":1776802946000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S152500162400217X"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,6]]},"references-count":40,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2024,6]]}},"alternative-id":["S152500162400217X"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2024.04.001","relation":{"has-preprint":[{"id-type":"doi","id":"10.1101\/2024.03.05.583596","asserted-by":"object"}]},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2024,6]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"TALEN-mediated intron editing of HSPCs enables transgene expression restricted to the myeloid lineage","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2024.04.001","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2024 The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}]}}