{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,10]],"date-time":"2026-07-10T15:41:16Z","timestamp":1783698076979,"version":"3.55.0"},"reference-count":71,"publisher":"Elsevier BV","issue":"6","license":[{"start":{"date-parts":[[2025,10,1]],"date-time":"2025-10-01T00:00:00Z","timestamp":1759276800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2025,10,1]],"date-time":"2025-10-01T00:00:00Z","timestamp":1759276800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,9,4]],"date-time":"2025-09-04T00:00:00Z","timestamp":1756944000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["82222030"],"award-info":[{"award-number":["82222030"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100012166","name":"National Key Research and Development Program of China","doi-asserted-by":"publisher","award":["2024YFC3406704"],"award-info":[{"award-number":["2024YFC3406704"]}],"id":[{"id":"10.13039\/501100012166","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100012166","name":"National Key Research and Development Program of China","doi-asserted-by":"publisher","award":["2023YFC3403301"],"award-info":[{"award-number":["2023YFC3403301"]}],"id":[{"id":"10.13039\/501100012166","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100013365","name":"West China Hospital, Sichuan University","doi-asserted-by":"publisher","award":["ZYYC23009"],"award-info":[{"award-number":["ZYYC23009"]}],"id":[{"id":"10.13039\/501100013365","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["neurotherapeuticsjournal.org","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Neurotherapeutics"],"published-print":{"date-parts":[[2025,10]]},"DOI":"10.1016\/j.neurot.2025.e00741","type":"journal-article","created":{"date-parts":[[2025,9,23]],"date-time":"2025-09-23T04:44:04Z","timestamp":1758602644000},"page":"e00741","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":2,"title":["Rescue of neurologic disease in mucopolysaccharidosis type II mice via AAV-mediated liver delivery of brain-penetrating iduronate-2-sulfatase"],"prefix":"10.1016","volume":"22","author":[{"given":"Xiu","family":"Jin","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Qin","family":"Ye","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaoyi","family":"Wu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jing","family":"Su","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Li","family":"Song","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jiamei","family":"Fu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Qiuxia","family":"Xu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Min","family":"Luo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fanfei","family":"Liu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chengda","family":"Ren","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ming","family":"Hu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Man","family":"Liu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yifang","family":"An","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Qiqi","family":"Li","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Manjun","family":"Li","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yang","family":"Yang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.neurot.2025.e00741_bib1","doi-asserted-by":"crossref","first-page":"7888","DOI":"10.3390\/ijms22157888","article-title":"Differences in MPS I and MPS II disease manifestations","volume":"22","author":"Hampe","year":"2021","journal-title":"Int J Mol Sci"},{"key":"10.1016\/j.neurot.2025.e00741_bib2","doi-asserted-by":"crossref","first-page":"4854","DOI":"10.3390\/ijms23094854","article-title":"Current and future treatment of mucopolysaccharidosis (MPS) type II: is brain-targeted stem cell gene therapy the solution for this devastating disorder?","volume":"23","author":"Horgan","year":"2022","journal-title":"Int J Mol Sci"},{"key":"10.1016\/j.neurot.2025.e00741_bib3","doi-asserted-by":"crossref","first-page":"267","DOI":"10.1007\/s00431-007-0635-4","article-title":"Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy","volume":"167","author":"Wraith","year":"2008","journal-title":"Eur J Pediatr"},{"key":"10.1016\/j.neurot.2025.e00741_bib4","doi-asserted-by":"crossref","first-page":"207","DOI":"10.1186\/s13052-024-01769-9","article-title":"The diagnosis and management of mucopolysaccharidosis type II","volume":"50","author":"Mao","year":"2024","journal-title":"Ital J Pediatr"},{"key":"10.1016\/j.neurot.2025.e00741_bib5","doi-asserted-by":"crossref","first-page":"18","DOI":"10.1016\/j.ymgme.2009.08.006","article-title":"Japan Elaprase Treatment (JET) study: idursulfase enzyme replacement therapy in adult patients with attenuated Hunter syndrome (Mucopolysaccharidosis II, MPS II)","volume":"99","author":"Okuyama","year":"2010","journal-title":"Mol Genet Metabol"},{"key":"10.1016\/j.neurot.2025.e00741_bib6","doi-asserted-by":"crossref","first-page":"473","DOI":"10.1007\/s10545-008-0878-x","article-title":"Neurological findings in Hunter disease: pathology and possible therapeutic effects reviewed","volume":"31","author":"Al","year":"2008","journal-title":"J Inherit Metab Dis"},{"key":"10.1016\/j.neurot.2025.e00741_bib7","first-page":"65","article-title":"Enzymatic replacement therapy for Hunter disease: up to 9 years experience with 17 patients","volume":"3","author":"Parini","year":"2015","journal-title":"Mol Genet Metab Rep"},{"key":"10.1016\/j.neurot.2025.e00741_bib8","doi-asserted-by":"crossref","first-page":"2975","DOI":"10.3390\/ijms21082975","article-title":"Intravenous enzyme replacement therapy in mucopolysaccharidoses: clinical effectiveness and limitations","volume":"21","author":"Parini","year":"2020","journal-title":"Int J Mol Sci"},{"key":"10.1016\/j.neurot.2025.e00741_bib9","doi-asserted-by":"crossref","first-page":"120","DOI":"10.1186\/s13052-018-0562-1","article-title":"Enzyme replacement therapy: efficacy and limitations","volume":"44","author":"Concolino","year":"2018","journal-title":"Ital J Pediatr"},{"key":"10.1016\/j.neurot.2025.e00741_bib10","doi-asserted-by":"crossref","first-page":"450","DOI":"10.1016\/j.molmed.2003.08.004","article-title":"Significance of immune response to enzyme-replacement therapy for patients with a lysosomal storage disorder","volume":"9","author":"Brooks","year":"2003","journal-title":"Trends Mol Med"},{"key":"10.1016\/j.neurot.2025.e00741_bib11","doi-asserted-by":"crossref","first-page":"72","DOI":"10.1186\/1750-1172-6-72","article-title":"Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease","volume":"6","author":"Scarpa","year":"2011","journal-title":"Orphanet J Rare Dis"},{"key":"10.1016\/j.neurot.2025.e00741_bib12","doi-asserted-by":"crossref","first-page":"456","DOI":"10.1016\/j.ymthe.2018.12.005","article-title":"Iduronate-2-Sulfatase with anti-human transferrin receptor antibody for neuropathic mucopolysaccharidosis II: a phase 1\/2 trial","volume":"27","author":"Okuyama","year":"2019","journal-title":"Mol Ther"},{"key":"10.1016\/j.neurot.2025.e00741_bib13","doi-asserted-by":"crossref","first-page":"671","DOI":"10.1016\/j.ymthe.2020.09.039","article-title":"A phase 2\/3 trial of pabinafusp Alfa, IDS fused with anti-human transferrin receptor antibody, targeting neurodegeneration in MPS-II","volume":"29","author":"Okuyama","year":"2021","journal-title":"Mol Ther"},{"key":"10.1016\/j.neurot.2025.e00741_bib14","doi-asserted-by":"crossref","first-page":"2317","DOI":"10.1002\/bit.25289","article-title":"Insulin receptor antibody-iduronate 2-sulfatase fusion protein: pharmacokinetics, anti-drug antibody, and safety pharmacology in Rhesus monkeys","volume":"111","author":"Boado","year":"2014","journal-title":"Biotechnol Bioeng"},{"key":"10.1016\/j.neurot.2025.e00741_bib15","doi-asserted-by":"crossref","first-page":"1741","DOI":"10.1021\/bc400319d","article-title":"Blood-brain barrier molecular trojan horse enables imaging of brain uptake of radioiodinated recombinant protein in the rhesus monkey","volume":"24","author":"Boado","year":"2013","journal-title":"Bioconjug Chem"},{"key":"10.1016\/j.neurot.2025.e00741_bib16","doi-asserted-by":"crossref","first-page":"145","DOI":"10.1016\/S0022-3476(96)70202-0","article-title":"Bone marrow transplantation in Hunter syndrome","volume":"129","author":"McKinnis","year":"1996","journal-title":"J Pediatr"},{"key":"10.1016\/j.neurot.2025.e00741_bib17","doi-asserted-by":"crossref","first-page":"229","DOI":"10.1038\/sj.bmt.1703839","article-title":"Working Party on Inborn Errors EBMTG. Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines","volume":"31","author":"Peters","year":"2003","journal-title":"Bone Marrow Transplant"},{"key":"10.1016\/j.neurot.2025.e00741_bib18","doi-asserted-by":"crossref","first-page":"4894","DOI":"10.1182\/blood.V87.11.4894.bloodjournal87114894","article-title":"Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome","volume":"87","author":"Peters","year":"1996","journal-title":"Blood"},{"key":"10.1016\/j.neurot.2025.e00741_bib19","series-title":"Seminars in hematology","first-page":"59","article-title":"Transplant outcomes in mucopolysaccharidoses","author":"Prasad","year":"2010"},{"key":"10.1016\/j.neurot.2025.e00741_bib20","doi-asserted-by":"crossref","first-page":"79","DOI":"10.1002\/ana.1098","article-title":"Brain magnetic resonance imaging in 23 patients with mucopolysaccharidoses and the effect of bone marrow transplantation","volume":"50","author":"Seto","year":"2001","journal-title":"Ann Neurol"},{"key":"10.1016\/j.neurot.2025.e00741_bib21","doi-asserted-by":"crossref","first-page":"638","DOI":"10.1023\/A:1005525931994","article-title":"Long-term follow-up following bone marrow transplantation for Hunter disease","volume":"22","author":"Vellodi","year":"1999","journal-title":"J Inherit Metab Dis"},{"issue":"Suppl 5","key":"10.1016\/j.neurot.2025.e00741_bib22","doi-asserted-by":"crossref","first-page":"v49","DOI":"10.1093\/rheumatology\/ker396","article-title":"Therapy for the mucopolysaccharidoses","volume":"50","author":"Valayannopoulos","year":"2011","journal-title":"Rheumatology (Oxford)"},{"key":"10.1016\/j.neurot.2025.e00741_bib23","doi-asserted-by":"crossref","first-page":"231","DOI":"10.1146\/annurev-med-043021-033013","article-title":"Adeno-associated virus gene therapy for hemophilia","volume":"74","author":"Samelson-Jones","year":"2023","journal-title":"Annu Rev Med"},{"key":"10.1016\/j.neurot.2025.e00741_bib24","doi-asserted-by":"crossref","first-page":"286","DOI":"10.1016\/j.omtm.2023.03.014","article-title":"Gene therapy for cross-correction of somatic organs and the CNS in mucopolysaccharidosis II in rodents and non-human primates","volume":"29","author":"Chen","year":"2023","journal-title":"Mol Ther Methods Clin Dev"},{"key":"10.1016\/j.neurot.2025.e00741_bib25","doi-asserted-by":"crossref","first-page":"1225","DOI":"10.1093\/hmg\/ddl038","article-title":"Correction of Hunter syndrome in the MPSII mouse model by AAV2\/8-mediated gene delivery","volume":"15","author":"Cardone","year":"2006","journal-title":"Hum Mol Genet"},{"key":"10.1016\/j.neurot.2025.e00741_bib26","doi-asserted-by":"crossref","first-page":"2074","DOI":"10.1177\/0271678X18772998","article-title":"A peptide derived from melanotransferrin delivers a protein-based interleukin 1 receptor antagonist across the BBB and ameliorates neuropathic pain in a preclinical model","volume":"39","author":"Thom","year":"2019","journal-title":"J Cerebr Blood Flow Metabol"},{"key":"10.1016\/j.neurot.2025.e00741_bib27","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.neurot.2025.e00741_bib28","doi-asserted-by":"crossref","first-page":"1259","DOI":"10.1089\/hum.2010.055","article-title":"Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale","volume":"21","author":"Lock","year":"2010","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.neurot.2025.e00741_bib29","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\u2013directed, AAV9-mediated iduronate sulfatase gene transfer","volume":"28","author":"Laoharawee","year":"2017","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.neurot.2025.e00741_bib30","doi-asserted-by":"crossref","first-page":"334","DOI":"10.1038\/nbt.3469","article-title":"A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice","volume":"34","author":"Yang","year":"2016","journal-title":"Nat Biotechnol"},{"key":"10.1016\/j.neurot.2025.e00741_bib31","doi-asserted-by":"crossref","first-page":"370","DOI":"10.1016\/j.omtm.2022.04.010","article-title":"Liver-directed gene therapy corrects neurologic disease in a murine model of mucopolysaccharidosis type I-Hurler","volume":"25","author":"Jin","year":"2022","journal-title":"Mol Ther Methods Clin Dev"},{"key":"10.1016\/j.neurot.2025.e00741_bib32","doi-asserted-by":"crossref","first-page":"e2389","DOI":"10.21769\/BioProtoc.2389","article-title":"Delayed-matching-to-place task in a dry maze to measure spatial working memory in mice","volume":"7","author":"Feng","year":"2017","journal-title":"Bio Protoc"},{"key":"10.1016\/j.neurot.2025.e00741_bib33","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.omtm.2021.04.017","article-title":"Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia","volume":"22","author":"Wang","year":"2021","journal-title":"Mol Ther Methods Clin Dev"},{"key":"10.1016\/j.neurot.2025.e00741_bib34","doi-asserted-by":"crossref","first-page":"97","DOI":"10.1038\/s41551-019-0501-5","article-title":"Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses","volume":"4","author":"Levy","year":"2020","journal-title":"Nat Biomed Eng"},{"key":"10.1016\/j.neurot.2025.e00741_bib35","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0035787","article-title":"Neuropathology in mouse models of mucopolysaccharidosis type I, IIIA and IIIB","volume":"7","author":"Wilkinson","year":"2012","journal-title":"PLoS One"},{"key":"10.1016\/j.neurot.2025.e00741_bib36","doi-asserted-by":"crossref","first-page":"142","DOI":"10.1002\/brb3.41","article-title":"Thy1-hAPPLond\/Swe+ mouse model of Alzheimer's disease displays broad behavioral deficits in sensorimotor, cognitive and social function","volume":"2","author":"Faizi","year":"2012","journal-title":"Brain Behav"},{"key":"10.1016\/j.neurot.2025.e00741_bib37","doi-asserted-by":"crossref","first-page":"6","DOI":"10.1186\/s43556-023-00120-8","article-title":"In vivo adenine base editing corrects newborn murine model of Hurler syndrome","volume":"4","author":"Su","year":"2023","journal-title":"Mol Biomed"},{"key":"10.1016\/j.neurot.2025.e00741_bib38","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0172435","article-title":"Identification of age-dependent motor and neuropsychological behavioural abnormalities in a mouse model of Mucopolysaccharidosis Type II","volume":"12","author":"Gleitz","year":"2017","journal-title":"PLoS One"},{"key":"10.1016\/j.neurot.2025.e00741_bib39","doi-asserted-by":"crossref","first-page":"290","DOI":"10.1007\/s00380-018-1242-1","article-title":"Aortic root dilatation in patients with mucopolysaccharidoses and the impact of enzyme replacement therapy","volume":"34","author":"Poswar","year":"2019","journal-title":"Heart Vessel"},{"key":"10.1016\/j.neurot.2025.e00741_bib40","doi-asserted-by":"crossref","first-page":"2028","DOI":"10.1038\/mt.2014.152","article-title":"Normalization and improvement of CNS deficits in mice with Hurler syndrome after long-term peripheral delivery of BBB-targeted iduronidase","volume":"22","author":"El-Amouri","year":"2014","journal-title":"Mol Ther"},{"key":"10.1016\/j.neurot.2025.e00741_bib41","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtm.2023.101149","article-title":"Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II","volume":"31","author":"Catalano","year":"2023","journal-title":"Mol Ther Methods Clin Dev"},{"key":"10.1016\/j.neurot.2025.e00741_bib42","doi-asserted-by":"crossref","first-page":"675","DOI":"10.1002\/emmm.201202083","article-title":"A highly secreted sulphamidase engineered to cross the blood-brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA","volume":"5","author":"Sorrentino","year":"2013","journal-title":"EMBO Mol Med"},{"key":"10.1016\/j.neurot.2025.e00741_bib43","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1089\/hum.2018.015","article-title":"Severe toxicity in nonhuman primates and piglets following high-dose intravenous administration of an adeno-associated virus vector expressing human SMN","volume":"29","author":"Hinderer","year":"2018","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.neurot.2025.e00741_bib44","doi-asserted-by":"crossref","first-page":"952","DOI":"10.1016\/j.ymthe.2024.02.002","article-title":"High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury","volume":"32","author":"Hordeaux","year":"2024","journal-title":"Mol Ther"},{"key":"10.1016\/j.neurot.2025.e00741_bib45","doi-asserted-by":"crossref","first-page":"842","DOI":"10.1089\/hum.2022.29216.bfs","article-title":"Novartis confirms deaths of two patients treated with gene therapy Zolgensma","volume":"33","author":"AJHGT","year":"2022","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.neurot.2025.e00741_bib46","doi-asserted-by":"crossref","first-page":"870","DOI":"10.1172\/JCI79213","article-title":"Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy","volume":"125","author":"Chandler","year":"2015","journal-title":"J Clin Investig"},{"key":"10.1016\/j.neurot.2025.e00741_bib47","doi-asserted-by":"crossref","first-page":"1343","DOI":"10.1038\/sj.gt.3301541","article-title":"Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors","volume":"8","author":"Donsante","year":"2001","journal-title":"Gene Ther"},{"key":"10.1016\/j.neurot.2025.e00741_bib48","doi-asserted-by":"crossref","first-page":"34","DOI":"10.1016\/j.ymthe.2006.03.008","article-title":"Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver","volume":"14","author":"Bell","year":"2006","journal-title":"Mol Ther"},{"key":"10.1016\/j.neurot.2025.e00741_bib49","doi-asserted-by":"crossref","first-page":"383","DOI":"10.1016\/j.omtm.2019.10.016","article-title":"Pathogenesis of hepatic tumors following gene therapy in murine and canine models of glycogen storage disease","volume":"15","author":"Kang","year":"2019","journal-title":"Mol Ther Methods Clin Dev"},{"key":"10.1016\/j.neurot.2025.e00741_bib50","doi-asserted-by":"crossref","first-page":"680","DOI":"10.1016\/j.ymthe.2020.10.018","article-title":"Liver injury increases the incidence of HCC following AAV gene therapy in mice","volume":"29","author":"Dalwadi","year":"2021","journal-title":"Mol Ther"},{"key":"10.1016\/j.neurot.2025.e00741_bib51","doi-asserted-by":"crossref","first-page":"3311","DOI":"10.1182\/blood-2010-08-302729","article-title":"Assessing the potential for AAV vector genotoxicity in a murine model","volume":"117","author":"Li","year":"2011","journal-title":"Blood"},{"key":"10.1016\/j.neurot.2025.e00741_bib52","doi-asserted-by":"crossref","first-page":"244","DOI":"10.1160\/TH13-02-0154","article-title":"Incorporation of the factor IX Padua mutation into FIX-Triple improves clotting activity in vitro and in vivo","volume":"110","author":"Kao","year":"2013","journal-title":"Thromb Haemost"},{"key":"10.1016\/j.neurot.2025.e00741_bib53","doi-asserted-by":"crossref","first-page":"327","DOI":"10.1016\/j.omtm.2018.07.005","article-title":"Targeting root cause by systemic scAAV9-hIDS gene delivery: functional correction and reversal of severe MPS II in mice","volume":"10","author":"Fu","year":"2018","journal-title":"Mol Ther Methods Clin Dev"},{"key":"10.1016\/j.neurot.2025.e00741_bib54","doi-asserted-by":"crossref","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.neurot.2025.e00741_bib55","doi-asserted-by":"crossref","DOI":"10.3389\/fnins.2021.596976","article-title":"Discovery of a highly conserved peptide in the iron transporter melanotransferrin that traverses an intact blood brain barrier and localizes in neural cells","volume":"15","author":"Singh","year":"2021","journal-title":"Front Neurosci"},{"key":"10.1016\/j.neurot.2025.e00741_bib56","doi-asserted-by":"crossref","first-page":"924","DOI":"10.1046\/j.1471-4159.2002.01201.x","article-title":"High transcytosis of melanotransferrin (P97) across the blood\u2013brain barrier","volume":"83","author":"Demeule","year":"2002","journal-title":"J Neurochem"},{"key":"10.1016\/j.neurot.2025.e00741_bib57","doi-asserted-by":"crossref","first-page":"1723","DOI":"10.1182\/blood-2003-01-0166","article-title":"Regulation of plasminogen activation: a role for melanotransferrin (p97) in cell migration","volume":"102","author":"Demeule","year":"2003","journal-title":"Blood"},{"key":"10.1016\/j.neurot.2025.e00741_bib58","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0249686","article-title":"Two peptides targeting endothelial receptors are internalized into murine brain endothelial cells","volume":"16","author":"Hudecz","year":"2021","journal-title":"PLoS One"},{"key":"10.1016\/j.neurot.2025.e00741_bib59","doi-asserted-by":"crossref","first-page":"14777","DOI":"10.1073\/pnas.0506892102","article-title":"Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII","volume":"102","author":"Vogler","year":"2005","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1016\/j.neurot.2025.e00741_bib60","doi-asserted-by":"crossref","first-page":"116","DOI":"10.1016\/j.ymgme.2013.09.008","article-title":"High-dose enzyme replacement therapy in murine Hurler syndrome","volume":"111","author":"Ou","year":"2014","journal-title":"Mol Genet Metabol"},{"key":"10.1016\/j.neurot.2025.e00741_bib61","doi-asserted-by":"crossref","first-page":"1902","DOI":"10.1073\/pnas.252784899","article-title":"Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB","volume":"100","author":"Ohmi","year":"2003","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1016\/j.neurot.2025.e00741_bib62","doi-asserted-by":"crossref","first-page":"612","DOI":"10.1002\/jnr.21134","article-title":"Cytokines, neurotrophins, and oxidative stress in brain disease from mucopolysaccharidosis IIIB","volume":"85","author":"Villani","year":"2007","journal-title":"J Neurosci Res"},{"key":"10.1016\/j.neurot.2025.e00741_bib63","doi-asserted-by":"crossref","first-page":"336","DOI":"10.1093\/brain\/awu355","article-title":"Neuroinflammation, mitochondrial defects and neurodegeneration in mucopolysaccharidosis III type C mouse model","volume":"138","author":"Martins","year":"2015","journal-title":"Brain"},{"key":"10.1016\/j.neurot.2025.e00741_bib64","doi-asserted-by":"crossref","first-page":"118","DOI":"10.1002\/(SICI)1098-1063(1999)9:2<118::AID-HIPO4>3.0.CO;2-8","article-title":"Delay-dependent impairment of a matching-to-place task with chronic and intrahippocampal infusion of the NMDA-antagonist D-AP5","volume":"9","author":"Steele","year":"1999","journal-title":"Hippocampus"},{"issue":"49-79","key":"10.1016\/j.neurot.2025.e00741_bib65","first-page":"C1","article-title":"Hippocampal-neocortical interactions in memory formation, consolidation, and reconsolidation","volume":"61","author":"Wang","year":"2010","journal-title":"Annu Rev Psychol"},{"key":"10.1016\/j.neurot.2025.e00741_bib66","doi-asserted-by":"crossref","first-page":"1057","DOI":"10.1016\/j.neuron.2012.12.002","article-title":"The role of medial prefrontal cortex in memory and decision making","volume":"76","author":"Euston","year":"2012","journal-title":"Neuron"},{"key":"10.1016\/j.neurot.2025.e00741_bib67","doi-asserted-by":"crossref","first-page":"261","DOI":"10.1016\/j.omtm.2020.09.012","article-title":"Ex vivo gene therapy treats bone complications of mucopolysaccharidosis type II mouse models through bone remodeling reactivation","volume":"19","author":"Wada","year":"2020","journal-title":"Mol Ther Methods Clin Dev"},{"key":"10.1016\/j.neurot.2025.e00741_bib68","doi-asserted-by":"crossref","first-page":"243","DOI":"10.1089\/hum.2023.002","article-title":"Establishment of the effectiveness of early versus late stem cell gene therapy in mucopolysaccharidosis II for treating central versus peripheral disease","volume":"35","author":"Mandolfo","year":"2024","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.neurot.2025.e00741_bib69","doi-asserted-by":"crossref","first-page":"256","DOI":"10.1089\/hum.2023.177","article-title":"Lentiviral gene therapy for mucopolysaccharidosis II with tagged iduronate 2-sulfatase prevents life-threatening pathology in peripheral tissues but fails to correct cartilage","volume":"35","author":"Catalano","year":"2024","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.neurot.2025.e00741_bib70","doi-asserted-by":"crossref","first-page":"90","DOI":"10.1002\/ajmg.a.34371","article-title":"Retrospective analysis of the clinical manifestations and survival of Korean patients with mucopolysaccharidosis type II: emphasis on the cardiovascular complication and mortality cases","volume":"158A","author":"Sohn","year":"2012","journal-title":"Am J Med Genet"},{"key":"10.1016\/j.neurot.2025.e00741_bib71","doi-asserted-by":"crossref","first-page":"50","DOI":"10.1186\/s13023-016-0425-z","article-title":"Severe tracheal and bronchial collapse in adults with type II mucopolysaccharidosis","volume":"11","author":"Rutten","year":"2016","journal-title":"Orphanet J Rare Dis"}],"container-title":["Neurotherapeutics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1878747925002193?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1878747925002193?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,3,10]],"date-time":"2026-03-10T16:12:10Z","timestamp":1773159130000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1878747925002193"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,10]]},"references-count":71,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2025,10]]}},"alternative-id":["S1878747925002193"],"URL":"https:\/\/doi.org\/10.1016\/j.neurot.2025.e00741","relation":{},"ISSN":["1878-7479"],"issn-type":[{"value":"1878-7479","type":"print"}],"subject":[],"published":{"date-parts":[[2025,10]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Rescue of neurologic disease in mucopolysaccharidosis type II mice via AAV-mediated liver delivery of brain-penetrating iduronate-2-sulfatase","name":"articletitle","label":"Article Title"},{"value":"Neurotherapeutics","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.neurot.2025.e00741","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 The Author(s). Published by Elsevier Inc. on behalf of American Society for Experimental NeuroTherapeutics.","name":"copyright","label":"Copyright"}],"article-number":"e00741"}}