{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,19]],"date-time":"2026-01-19T09:34:14Z","timestamp":1768815254168,"version":"3.49.0"},"reference-count":78,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2023,12,1]],"date-time":"2023-12-01T00:00:00Z","timestamp":1701388800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2023,12,1]],"date-time":"2023-12-01T00:00:00Z","timestamp":1701388800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2023,11,1]],"date-time":"2023-11-01T00:00:00Z","timestamp":1698796800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100003061","name":"Erasmus Medisch Centrum","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100003061","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003246","name":"Nederlandse Organisatie voor Wetenschappelijk Onderzoek","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100003246","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004243","name":"Prinses Beatrix Spierfonds","doi-asserted-by":"publisher","award":["20210367"],"award-info":[{"award-number":["20210367"]}],"id":[{"id":"10.13039\/501100004243","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004243","name":"Prinses Beatrix Spierfonds","doi-asserted-by":"publisher","award":["DR-2021-00386"],"award-info":[{"award-number":["DR-2021-00386"]}],"id":[{"id":"10.13039\/501100004243","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004243","name":"Prinses Beatrix Spierfonds","doi-asserted-by":"publisher","award":["W.OP20-04"],"award-info":[{"award-number":["W.OP20-04"]}],"id":[{"id":"10.13039\/501100004243","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100016062","name":"Metakids Foundation","doi-asserted-by":"publisher","award":["2018-083"],"award-info":[{"award-number":["2018-083"]}],"id":[{"id":"10.13039\/100016062","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100016062","name":"Metakids Foundation","doi-asserted-by":"publisher","award":["2021-094"],"award-info":[{"award-number":["2021-094"]}],"id":[{"id":"10.13039\/100016062","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100008358","name":"Brain Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100008358","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100022691","name":"National Weather Association","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100022691","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100010573","name":"Dioraphte Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100010573","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100022790","name":"Sophia Foundation","doi-asserted-by":"publisher","award":["S18-59"],"award-info":[{"award-number":["S18-59"]}],"id":[{"id":"10.13039\/100022790","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":[[2023,12]]},"DOI":"10.1016\/j.omtm.2023.101149","type":"journal-article","created":{"date-parts":[[2023,11,2]],"date-time":"2023-11-02T20:18:34Z","timestamp":1698956314000},"page":"101149","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":6,"special_numbering":"C","title":["Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II"],"prefix":"10.1016","volume":"31","author":[{"given":"Fabio","family":"Catalano","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Eva C.","family":"Vlaar","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Drosos","family":"Katsavelis","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Zina","family":"Dammou","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tessa F.","family":"Huizer","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jeroen C.","family":"van den Bosch","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marianne","family":"Hoogeveen-Westerveld","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hannerieke J.M.P.","family":"van den Hout","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Esmeralda","family":"Oussoren","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"George J.G.","family":"Ruijter","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gerben","family":"Schaaf","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Karin","family":"Pike-Overzet","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Frank J.T.","family":"Staal","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ans T.","family":"van der Ploeg","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7042-2482","authenticated-orcid":false,"given":"W.W.M. Pim","family":"Pijnappel","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2023.101149_bib1","doi-asserted-by":"crossref","first-page":"104","DOI":"10.1177\/003591571701001833","article-title":"A Rare Disease in Two Brothers","volume":"10","author":"Hunter","year":"1917","journal-title":"Proc. Roy. Soc. Med."},{"key":"10.1016\/j.omtm.2023.101149_bib2","doi-asserted-by":"crossref","first-page":"2134","DOI":"10.1073\/pnas.70.7.2134","article-title":"The defect in the Hunter syndrome: deficiency of sulfoiduronate sulfatase","volume":"70","author":"Bach","year":"1973","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2023.101149_bib3","doi-asserted-by":"crossref","first-page":"8531","DOI":"10.1073\/pnas.87.21.8531","article-title":"Hunter syndrome: isolation of an iduronate-2-sulfatase cDNA clone and analysis of patient DNA","volume":"87","author":"Wilson","year":"1990","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2023.101149_bib4","doi-asserted-by":"crossref","first-page":"773","DOI":"10.1006\/geno.1993.1406","article-title":"Sequence of the Human Iduronate 2-Sulfatase (IDS) Gene","volume":"17","author":"Wilson","year":"1993","journal-title":"Genomics"},{"key":"10.1016\/j.omtm.2023.101149_bib5","doi-asserted-by":"crossref","DOI":"10.3390\/ijms21041258","article-title":"Mucopolysaccharidosis Type II: One Hundred Years of Research, Diagnosis, and Treatment","volume":"21","author":"D\u02b9avanzo","year":"2020","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.omtm.2023.101149_bib6","first-page":"100816","article-title":"Real world long-term outcomes in patients with mucopolysaccharidosis type II: A retrospective cohort study","volume":"29","author":"Tomita","year":"2021","journal-title":"Mol. Genet. Metab. Rep."},{"key":"10.1016\/j.omtm.2023.101149_bib7","doi-asserted-by":"crossref","first-page":"456","DOI":"10.1186\/s13023-021-02052-4","article-title":"Evaluation of the long-term treatment effects of intravenous idursulfase in patients with mucopolysaccharidosis II (MPS II) using statistical modeling: data from the Hunter Outcome Survey (HOS)","volume":"16","author":"Muenzer","year":"2021","journal-title":"Orphanet J. Rare Dis."},{"key":"10.1016\/j.omtm.2023.101149_bib8","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.aay1163","article-title":"Brain delivery and activity of a lysosomal enzyme using a blood-brain barrier transport vehicle in mice","volume":"12","author":"Ullman","year":"2020","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtm.2023.101149_bib9","doi-asserted-by":"crossref","DOI":"10.1084\/jem.20211057","article-title":"Molecular architecture determines brain delivery of a transferrin receptor-targeted lysosomal enzyme","volume":"219","author":"Arguello","year":"2022","journal-title":"J.\u00a0Exp. Med."},{"key":"10.1016\/j.omtm.2023.101149_bib10","doi-asserted-by":"crossref","first-page":"626","DOI":"10.1089\/hum.2016.184","article-title":"Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System-Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer","volume":"28","author":"Laoharawee","year":"2017","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2023.101149_bib11","doi-asserted-by":"crossref","first-page":"e86696","DOI":"10.1172\/jci.insight.86696","article-title":"CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)","volume":"1","author":"Motas","year":"2016","journal-title":"JCI Insight"},{"key":"10.1016\/j.omtm.2023.101149_bib12","doi-asserted-by":"crossref","first-page":"68","DOI":"10.1016\/j.omtm.2018.06.004","article-title":"Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques","volume":"10","author":"Hordeaux","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2023.101149_bib13","doi-asserted-by":"crossref","first-page":"1260","DOI":"10.1089\/hum.2021.053","article-title":"Long-Term Follow-Up of Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy","volume":"32","author":"Bougn\u00e8res","year":"2021","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2023.101149_bib14","doi-asserted-by":"crossref","first-page":"2002","DOI":"10.1056\/NEJMoa2027675","article-title":"Autologous Ex\u00a0Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency","volume":"384","author":"Kohn","year":"2021","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2023.101149_bib15","doi-asserted-by":"crossref","first-page":"489","DOI":"10.1016\/j.ymthe.2020.11.020","article-title":"Update on Clinical Ex\u00a0Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases","volume":"29","author":"Tucci","year":"2021","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2023.101149_bib16","doi-asserted-by":"crossref","first-page":"372","DOI":"10.1016\/S0140-6736(21)02017-1","article-title":"Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1\/2 trial and expanded access","volume":"399","author":"Fumagalli","year":"2022","journal-title":"Lancet"},{"key":"10.1016\/j.omtm.2023.101149_bib17","doi-asserted-by":"crossref","first-page":"1304","DOI":"10.1182\/blood.2020010260","article-title":"Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency","volume":"138","author":"Reinhardt","year":"2021","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2023.101149_bib18","doi-asserted-by":"crossref","first-page":"e1701211","DOI":"10.1126\/sciadv.1701211","article-title":"Intracerebroventricular delivery of hematopoietic progenitors results in rapid and robust engraftment of microglia-like cells","volume":"3","author":"Capotondo","year":"2017","journal-title":"Sci. Adv."},{"key":"10.1016\/j.omtm.2023.101149_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.omtm.2023.101149_bib20","doi-asserted-by":"crossref","first-page":"357","DOI":"10.1089\/hum.2014.158","article-title":"Hematopoietic Stem Cell Gene Therapy Corrects Neuropathic Phenotype in Murine Model of Mucopolysaccharidosis Type II","volume":"26","author":"Wakabayashi","year":"2015","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2023.101149_bib21","doi-asserted-by":"crossref","first-page":"262","DOI":"10.1016\/j.ymgme.2020.06.007","article-title":"Efficient engraftment of genetically modified cells is necessary to ameliorate central nervous system involvement of murine model of mucopolysaccharidosis type II by hematopoietic stem cell targeted gene therapy","volume":"130","author":"Miwa","year":"2020","journal-title":"Mol. Genet. Metabol."},{"key":"10.1016\/j.omtm.2023.101149_bib22","doi-asserted-by":"crossref","first-page":"1279","DOI":"10.1089\/hum.2022.141","article-title":"Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex\u00a0Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells","volume":"33","author":"Smith","year":"2022","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2023.101149_bib23","doi-asserted-by":"crossref","DOI":"10.15252\/emmm.201708730","article-title":"Brain-targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms","volume":"10","author":"Gleitz","year":"2018","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.omtm.2023.101149_bib24","doi-asserted-by":"crossref","first-page":"818","DOI":"10.1126\/science.1171242","article-title":"Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy","volume":"326","author":"Cartier","year":"2009","journal-title":"Science"},{"key":"10.1016\/j.omtm.2023.101149_bib25","doi-asserted-by":"crossref","first-page":"109","DOI":"10.1016\/j.omtm.2022.09.010","article-title":"IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy","volume":"27","author":"Liang","year":"2022","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2023.101149_bib26","doi-asserted-by":"crossref","first-page":"306","DOI":"10.1016\/j.jconrel.2018.04.035","article-title":"A novel peptide ligand RAP12 of LRP1 for glioma targeted drug delivery","volume":"279","author":"Ruan","year":"2018","journal-title":"J.\u00a0Contr. Release"},{"key":"10.1016\/j.omtm.2023.101149_bib27","doi-asserted-by":"crossref","first-page":"3122","DOI":"10.1523\/JNEUROSCI.4785-13.2014","article-title":"Comparison of Five Peptide Vectors for Improved Brain Delivery of the Lysosomal Enzyme Arylsulfatase A","volume":"34","author":"B\u00f6ckenhoff","year":"2014","journal-title":"J.\u00a0Neurosci."},{"key":"10.1016\/j.omtm.2023.101149_bib28","doi-asserted-by":"crossref","first-page":"10182","DOI":"10.1073\/pnas.95.17.10182","article-title":"Consistent, persistent expression from modified retroviral vectors in murine hematopoietic stem cells","volume":"95","author":"Robbins","year":"1998","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2023.101149_bib29","doi-asserted-by":"crossref","first-page":"425","DOI":"10.1042\/bj3090425","article-title":"Processing of iduronate 2-sulphatase in human fibroblasts","volume":"309","author":"Froissart","year":"1995","journal-title":"Biochem. J."},{"key":"10.1016\/j.omtm.2023.101149_bib30","first-page":"A427","article-title":"Targeted disruption of the mouse iduronate sulfatase gene","volume":"65","author":"Chen","year":"1999","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.omtm.2023.101149_bib31","doi-asserted-by":"crossref","first-page":"924","DOI":"10.1007\/s10545-007-0641-8","article-title":"The characterization of a murine model of mucopolysaccharidosis II (Hunter syndrome)","volume":"30","author":"Garcia","year":"2007","journal-title":"J.\u00a0Inherit. Metab. Dis."},{"key":"10.1016\/j.omtm.2023.101149_bib32","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1016\/j.ymgme.2018.07.013","article-title":"Evaluation of cerebrospinal fluid heparan sulfate as a biomarker of neuropathology in a murine model of mucopolysaccharidosis type II using high-sensitivity LC\/MS\/MS","volume":"125","author":"Tanaka","year":"2018","journal-title":"Mol. Genet. Metabol."},{"key":"10.1016\/j.omtm.2023.101149_bib33","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.omtm.2023.101149_bib34","doi-asserted-by":"crossref","first-page":"2236","DOI":"10.3390\/cells10092236","article-title":"Beyond Activation: Characterizing Microglial Functional Phenotypes","volume":"10","author":"Lier","year":"2021","journal-title":"Cells"},{"key":"10.1016\/j.omtm.2023.101149_bib35","doi-asserted-by":"crossref","first-page":"1233158","DOI":"10.1126\/science.1233158","article-title":"Lentiviral Hematopoietic Stem Cell Gene Therapy Benefi ts Metachromatic Leukodystrophy","volume":"341","author":"Biffi","year":"2013","journal-title":"Science"},{"key":"10.1016\/j.omtm.2023.101149_bib36","doi-asserted-by":"crossref","first-page":"363","DOI":"10.1007\/s00401-014-1330-y","article-title":"The role of microglia in human disease: therapeutic tool or target?","volume":"128","author":"Cartier","year":"2014","journal-title":"Acta Neuropathol."},{"key":"10.1016\/j.omtm.2023.101149_bib37","doi-asserted-by":"crossref","first-page":"2857","DOI":"10.1172\/JCI30230","article-title":"Microglia: a cellular vehicle for CNS gene therapy","volume":"116","author":"Neumann","year":"2006","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2023.101149_bib38","doi-asserted-by":"crossref","first-page":"9945","DOI":"10.1126\/scitranslmed.abl9945","article-title":"Treatment of a genetic brain disease by CNS-wide microglia replacement","volume":"14","author":"Shibuya","year":"2022","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtm.2023.101149_bib39","doi-asserted-by":"crossref","first-page":"33","DOI":"10.3389\/fncel.2022.816439","article-title":"Microglia and Microglia-Like Cells: Similar but Different","volume":"16","author":"Cuadros","year":"2022","journal-title":"Front. Cell. Neurosci."},{"key":"10.1016\/j.omtm.2023.101149_bib40","doi-asserted-by":"crossref","first-page":"15018","DOI":"10.1073\/pnas.1205858109","article-title":"Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation","volume":"109","author":"Capotondo","year":"2012","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2023.101149_bib41","doi-asserted-by":"crossref","first-page":"1428","DOI":"10.1074\/jbc.M112.438663","article-title":"Glycosylation-independent lysosomal targeting of acid \u03b1-glucosidase enhances muscle glycogen clearance in pompe mice","volume":"288","author":"Maga","year":"2013","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2023.101149_bib42","doi-asserted-by":"crossref","first-page":"56","DOI":"10.1016\/j.omtm.2019.05.008","article-title":"Differential Uptake of NAGLU-IGF2 and Unmodified NAGLU in Cellular Models of Sanfilippo Syndrome Type B","volume":"14","author":"Prill","year":"2019","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2023.101149_bib43","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0207836","article-title":"BMN 250, a fusion of lysosomal alpha-N-acetylglucosaminidase with IGF2, exhibits different patterns of cellular uptake into critical cell types of Sanfilippo syndrome B disease pathogenesis","volume":"14","author":"Yogalingam","year":"2019","journal-title":"PLoS One"},{"key":"10.1016\/j.omtm.2023.101149_bib44","doi-asserted-by":"crossref","first-page":"1753","DOI":"10.1210\/endo.135.5.7525251","article-title":"Insulin-like growth factors cross the blood-brain barrier","volume":"135","author":"Reinhardt","year":"1994","journal-title":"Endocrinology"},{"key":"10.1016\/j.omtm.2023.101149_bib45","doi-asserted-by":"crossref","first-page":"136","DOI":"10.1016\/S0026-0495(98)90007-5","article-title":"Human blood-brain barrier insulin-like growth factor receptor","volume":"37","author":"Duffy","year":"1988","journal-title":"Metabolism"},{"key":"10.1016\/j.omtm.2023.101149_bib46","doi-asserted-by":"crossref","first-page":"225","DOI":"10.1038\/nrn3209","article-title":"The many faces of insulin-like peptide signalling in the brain","volume":"13","author":"Fernandez","year":"2012","journal-title":"Nat. Rev. Neurosci."},{"key":"10.1016\/j.omtm.2023.101149_bib47","doi-asserted-by":"crossref","first-page":"2148","DOI":"10.1212\/WNL.0b013e3182752eef","article-title":"Insulin-like growth factors in the brain and their potential clinical implications","volume":"79","author":"Benarroch","year":"2012","journal-title":"Neurology"},{"key":"10.1016\/j.omtm.2023.101149_bib48","doi-asserted-by":"crossref","first-page":"1261","DOI":"10.1038\/mt.2008.84","article-title":"Mannose 6-Phosphate Receptor\u2013mediated Transport of Sulfamidase Across the Blood\u2013brain Barrier in the Newborn Mouse","volume":"16","author":"Urayama","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2023.101149_bib49","doi-asserted-by":"crossref","first-page":"12658","DOI":"10.1073\/pnas.0405042101","article-title":"Developmentally regulated mannose 6-phosphate receptor-mediated transport of a lysosomal enzyme across the blood-brain barrier","volume":"101","author":"Urayama","year":"2004","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2023.101149_bib50","doi-asserted-by":"crossref","first-page":"228","DOI":"10.1111\/cen3.12037","article-title":"Toward the successful delivery of lysosomal enzymes across the blood-brain barrier","volume":"4","author":"Urayama","year":"2013","journal-title":"Clin. Exp. Neuroimmunol."},{"key":"10.1016\/j.omtm.2023.101149_bib51","doi-asserted-by":"crossref","first-page":"2373","DOI":"10.1021\/acs.biochem.7b01260","article-title":"Converting Insulin-like Growth Factors 1 and 2 into High-Affinity Ligands for Insulin Receptor Isoform A by the Introduction of an Evolutionarily Divergent Mutation","volume":"57","author":"Mach\u00e1\u010dkov\u00e1","year":"2018","journal-title":"Biochemistry"},{"key":"10.1016\/j.omtm.2023.101149_bib52","doi-asserted-by":"crossref","first-page":"2926","DOI":"10.1523\/JNEUROSCI.20-08-02926.2000","article-title":"Circulating insulin-like growth factor I mediates effects of exercise on the brain","volume":"20","author":"Carro","year":"2000","journal-title":"J.\u00a0Neurosci."},{"key":"10.1016\/j.omtm.2023.101149_bib53","doi-asserted-by":"crossref","first-page":"2276","DOI":"10.3390\/cells9102276","article-title":"Understanding IGF-II Action through Insights into Receptor Binding and Activation","volume":"9","author":"Blyth","year":"2020","journal-title":"Cells"},{"key":"10.1016\/j.omtm.2023.101149_bib54","doi-asserted-by":"crossref","first-page":"786","DOI":"10.1016\/j.str.2020.05.002","article-title":"How IGF-II Binds to the Human Type 1 Insulin-like Growth Factor Receptor","volume":"28","author":"Xu","year":"2020","journal-title":"Structure"},{"key":"10.1016\/j.omtm.2023.101149_bib55","doi-asserted-by":"crossref","first-page":"389","DOI":"10.1002\/bies.201400190","article-title":"Insulin\/receptor binding: The last piece of the puzzle?","volume":"37","author":"de Meyts","year":"2015","journal-title":"Bioessays"},{"key":"10.1016\/j.omtm.2023.101149_bib56","doi-asserted-by":"crossref","first-page":"14870","DOI":"10.1073\/pnas.1416660111","article-title":"Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB","volume":"111","author":"Kan","year":"2014","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2023.101149_bib57","doi-asserted-by":"crossref","first-page":"e0122453","DOI":"10.1371\/journal.pone.0122453","article-title":"Pharmacokinetics and Bioavailability of a Therapeutic Enzyme (Idursulfase) in Cynomolgus Monkeys after Intrathecal and Intravenous Administration","volume":"10","author":"Xie","year":"2015","journal-title":"PLoS One"},{"key":"10.1016\/j.omtm.2023.101149_bib58","doi-asserted-by":"crossref","first-page":"7328","DOI":"10.1021\/bi036208p","article-title":"Two Apolipoprotein E Mimetic Peptides, ApoE(130\u2212149) and ApoE(141\u2212155)2, Bind to LRP1","volume":"43","author":"Croy","year":"2004","journal-title":"Biochemistry"},{"key":"10.1016\/j.omtm.2023.101149_bib59","doi-asserted-by":"crossref","first-page":"4690","DOI":"10.1039\/C6CS00076B","article-title":"Blood-brain barrier shuttle peptides: an emerging paradigm for brain delivery","volume":"45","author":"Oller-Salvia","year":"2016","journal-title":"Chem. Soc. Rev."},{"key":"10.1016\/j.omtm.2023.101149_bib60","doi-asserted-by":"crossref","first-page":"16","DOI":"10.1186\/1750-1326-5-16","article-title":"ApoE mimetic peptide decreases A production in\u00a0vitro and in\u00a0vivo","volume":"5","author":"Minami","year":"2010","journal-title":"Mol. Neurodegener."},{"key":"10.1016\/j.omtm.2023.101149_bib61","doi-asserted-by":"crossref","first-page":"2999","DOI":"10.1073\/pnas.1222742110","article-title":"Engineering a lysosomal enzyme with a derivative of receptor-binding domain of apoE enables delivery across the blood-brain barrier","volume":"110","author":"Wang","year":"2013","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2023.101149_bib62","doi-asserted-by":"crossref","DOI":"10.1016\/j.expneurol.2022.114051","article-title":"Therapeutic potential of ApoE-mimetic peptides in CNS disorders: Current perspective","volume":"353","author":"Ahmed","year":"2022","journal-title":"Exp. Neurol."},{"key":"10.1016\/j.omtm.2023.101149_bib63","doi-asserted-by":"crossref","first-page":"2779","DOI":"10.1016\/j.bbagen.2018.08.020","article-title":"Quantification of Kinetic Rate Constants for Transcytosis of Polymeric Nanoparticle through Blood-Brain Barrier","volume":"1862","author":"Khan","year":"2018","journal-title":"Biochim. Biophys. Acta Gen. Subj."},{"key":"10.1016\/j.omtm.2023.101149_bib64","series-title":"Cellular, Tissue, and Gene Therapies Advisory Committee June 9-10, 2022 Meeting Presentation- Elivaldogene Autotemcel (Eli-Cel): BLA 125755 Clinical Considerations for Efficacy and Specific Safety in Early Cerebral Adrenoleukodystrophy","year":"2022"},{"key":"10.1016\/j.omtm.2023.101149_bib65","series-title":"Cellular, Tissue, and Gene Therapies Advisory Committee June 9, 2022- June 10, 2022 Meeting Briefing Document- FDA- 125755","year":"2022"},{"key":"10.1016\/j.omtm.2023.101149_bib67","series-title":"Cellular, Tissue, and Gene Therapies Advisory Committee June 9-10, 2022 Meeting Announcement","year":"2022"},{"key":"10.1016\/j.omtm.2023.101149_bib68","doi-asserted-by":"crossref","first-page":"1627","DOI":"10.1084\/jem.20180247","article-title":"Peripherally derived macrophages can engraft the brain independent of irradiation and maintain an identity distinct from microglia","volume":"215","author":"Cronk","year":"2018","journal-title":"J.\u00a0Exp. Med."},{"key":"10.1016\/j.omtm.2023.101149_bib69","doi-asserted-by":"crossref","first-page":"e20220857","DOI":"10.1084\/jem.20220857","article-title":"Engineering an inhibitor-resistant human CSF1R variant for microglia replacement","volume":"220","author":"Paul Chadarevian","year":"2022","journal-title":"J.\u00a0Exp. Med."},{"key":"10.1016\/j.omtm.2023.101149_bib70","doi-asserted-by":"crossref","first-page":"279","DOI":"10.1186\/s12974-020-01931-0","article-title":"Effects of long-term and brain-wide colonization of peripheral bone marrow-derived myeloid cells in the CNS","volume":"17","author":"Hohsfield","year":"2020","journal-title":"J.\u00a0Neuroinflammation"},{"key":"10.1016\/j.omtm.2023.101149_bib71","doi-asserted-by":"crossref","first-page":"868","DOI":"10.1038\/mt.2013.29","article-title":"Busulfan Conditioning Enhances Engraftment of Hematopoietic Donor-derived Cells in the Brain Compared With Irradiation","volume":"21","author":"Wilkinson","year":"2013","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2023.101149_bib72","doi-asserted-by":"crossref","first-page":"255","DOI":"10.1016\/j.ymgme.2009.05.006","article-title":"Localized donor cells in brain of a Hunter disease patient after cord blood stem cell transplantation","volume":"98","author":"Araya","year":"2009","journal-title":"Mol. Genet. Metabol."},{"key":"10.1016\/j.omtm.2023.101149_bib73","doi-asserted-by":"crossref","first-page":"71","DOI":"10.1111\/j.1651-2227.2002.tb03118.x","article-title":"Enzyme replacement therapy in mucopolysaccharidosis type II (Hunter syndrome): a preliminary report","volume":"91","author":"Muenzer","year":"2002","journal-title":"Acta Paediatr. Suppl."},{"key":"10.1016\/j.omtm.2023.101149_bib74","doi-asserted-by":"crossref","first-page":"666","DOI":"10.1016\/j.omtm.2020.03.016","article-title":"Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID","volume":"17","author":"Garcia-Perez","year":"2020","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2023.101149_bib75","doi-asserted-by":"crossref","first-page":"745","DOI":"10.1002\/psc.2570","article-title":"An apolipoprotein E mimetic peptide with activities against multidrug-resistant bacteria and immunomodulatory effects","volume":"19","author":"Wang","year":"2013","journal-title":"J.\u00a0Pept. Sci."},{"key":"10.1016\/j.omtm.2023.101149_bib76","doi-asserted-by":"crossref","first-page":"8463","DOI":"10.1128\/JVI.72.11.8463-8471.1998","article-title":"A Third-Generation Lentivirus Vector with a Conditional Packaging System","volume":"72","author":"Dull","year":"1998","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.omtm.2023.101149_bib77","doi-asserted-by":"crossref","first-page":"422","DOI":"10.1038\/s41431-020-00751-3","article-title":"A generic assay for the identification of splicing variants that induce nonsense-mediated decay in Pompe disease","volume":"29","author":"Bergsma","year":"2021","journal-title":"Eur. J. Hum. Genet."},{"key":"10.1016\/j.omtm.2023.101149_bib78","doi-asserted-by":"crossref","first-page":"520","DOI":"10.1016\/j.omtm.2022.04.016","article-title":"Lentiviral gene therapy prevents anti-human acid \u03b1-glucosidase antibody formation in murine Pompe disease","volume":"25","author":"Liang","year":"2022","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2023.101149_bib79","first-page":"675","article-title":"A fluorimetric enzyme assay for the diagnosis of MPS II (hunter disease)","volume":"24","author":"Voznyi","year":"2001"}],"container-title":["Molecular Therapy Methods & Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050123001882?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050123001882?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,8]],"date-time":"2025-11-08T15:20:08Z","timestamp":1762615208000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050123001882"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,12]]},"references-count":78,"alternative-id":["S2329050123001882"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2023.101149","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2023,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2023.101149","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2023 The Authors.","name":"copyright","label":"Copyright"}],"article-number":"101149"}}