{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,28]],"date-time":"2026-10-28T00:29:21Z","timestamp":1793147361292,"version":"4.5.0"},"reference-count":69,"publisher":"Elsevier BV","issue":"4","license":[{"start":{"date-parts":[[2024,12,1]],"date-time":"2024-12-01T00:00:00Z","timestamp":1733011200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2024,12,1]],"date-time":"2024-12-01T00:00:00Z","timestamp":1733011200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2024,10,25]],"date-time":"2024-10-25T00:00:00Z","timestamp":1729814400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100010661","name":"Horizon 2020 Framework Programme","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100010661","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":[[2024,12]]},"DOI":"10.1016\/j.omtm.2024.101363","type":"journal-article","created":{"date-parts":[[2024,10,30]],"date-time":"2024-10-30T07:43:26Z","timestamp":1730274206000},"page":"101363","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":10,"title":["Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I"],"prefix":"10.1016","volume":"32","author":[{"given":"Xiaoxia","family":"Shi","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giulia","family":"Bortolussi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fanny","family":"Collaud","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pierre-Romain","family":"Le Brun","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lysbeth ten","family":"Bloemendaal","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicolas","family":"Guerchet","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dirk","family":"Rudi de Waart","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pauline","family":"Sellier","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suzanne","family":"Duijst","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Philippe","family":"Veron","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Federico","family":"Mingozzi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Takashi Kei","family":"Kishimoto","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giuseppe","family":"Ronzitti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Piter","family":"Bosma","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9628-0494","authenticated-orcid":false,"given":"Andr\u00e9s F.","family":"Muro","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2024.101363_bib1","doi-asserted-by":"crossref","first-page":"17960","DOI":"10.1016\/S0021-9258(17)32403-1","article-title":"Bilirubin UDP-glucuronosyltransferase 1 is the only relevant bilirubin glucuronidating isoform in man","volume":"269","author":"Bosma","year":"1994","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2024.101363_bib2","doi-asserted-by":"crossref","first-page":"1923","DOI":"10.1002\/hep.30959","article-title":"Crigler-Najjar Syndrome Type 1: Pathophysiology, Natural History, and Therapeutic Frontier","volume":"71","author":"Strauss","year":"2020","journal-title":"Hepatology"},{"key":"10.1016\/j.omtm.2024.101363_bib3","doi-asserted-by":"crossref","DOI":"10.1111\/ctr.14219","article-title":"Outcome of liver transplantation and prevalence of liver fibrosis in Crigler-Najjar syndrome","volume":"35","author":"Schr\u00f6der","year":"2021","journal-title":"Clin. Transplant."},{"key":"10.1016\/j.omtm.2024.101363_bib4","doi-asserted-by":"crossref","first-page":"675","DOI":"10.1016\/j.jhep.2012.04.015","article-title":"Evolution of indications and results of liver transplantation in Europe. A report from the European Liver Transplant Registry (ELTR)","volume":"57","author":"Adam","year":"2012","journal-title":"J.\u00a0Hepatol."},{"key":"10.1016\/j.omtm.2024.101363_bib5","doi-asserted-by":"crossref","first-page":"425","DOI":"10.1080\/21678707.2018.1495558","article-title":"Advances in understanding disease mechanisms and potential treatments for Crigler\u2013Najjar syndrome","volume":"6","author":"Bortolussi","year":"2018","journal-title":"Expert Opin. Orphan Drugs"},{"key":"10.1016\/j.omtm.2024.101363_bib6","doi-asserted-by":"crossref","DOI":"10.1038\/mtm.2016.49","article-title":"A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome","volume":"3","author":"Ronzitti","year":"2016","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2024.101363_bib7","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1016\/j.omtm.2018.12.011","article-title":"Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome","volume":"12","author":"Collaud","year":"2019","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2024.101363_bib8","doi-asserted-by":"crossref","first-page":"844","DOI":"10.1089\/hum.2013.233","article-title":"Life-long correction of hyperbilirubinemia with a neonatal liver-specific AAV-mediated gene transfer in a lethal mouse model of Crigler-Najjar Syndrome","volume":"25","author":"Bortolussi","year":"2014","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib9","doi-asserted-by":"crossref","first-page":"1085","DOI":"10.1016\/j.ymthe.2006.01.014","article-title":"Adeno-associated Virus Vector Serotypes Mediate Sustained Correction of Bilirubin UDP Glucuronosyltransferase Deficiency in Rats","volume":"13","author":"Seppen","year":"2006","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib10","doi-asserted-by":"crossref","first-page":"620","DOI":"10.1056\/NEJMoa2214084","article-title":"Gene Therapy in Patients with the Crigler\u2013Najjar Syndrome","volume":"389","author":"D\u2019Antiga","year":"2023","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2024.101363_bib11","doi-asserted-by":"crossref","first-page":"1081","DOI":"10.1038\/mt.2008.72","article-title":"Gene delivery to the juvenile mouse liver using AAV2\/8 vectors","volume":"16","author":"Cunningham","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib12","doi-asserted-by":"crossref","first-page":"533","DOI":"10.1089\/hum.2011.183","article-title":"Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector","volume":"23","author":"Wang","year":"2012","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib13","doi-asserted-by":"crossref","first-page":"287","DOI":"10.1016\/j.omtm.2020.11.016","article-title":"Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients","volume":"20","author":"Shi","year":"2021","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2024.101363_bib14","doi-asserted-by":"crossref","first-page":"511","DOI":"10.1146\/annurev-virology-101416-041936","article-title":"Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape","volume":"4","author":"Mingozzi","year":"2017","journal-title":"Annu. Rev. Virol."},{"key":"10.1016\/j.omtm.2024.101363_bib15","doi-asserted-by":"crossref","first-page":"2215","DOI":"10.1056\/NEJMoa1708538","article-title":"Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant","volume":"377","author":"George","year":"2017","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2024.101363_bib16","doi-asserted-by":"crossref","first-page":"1994","DOI":"10.1056\/NEJMoa1407309","article-title":"Long-term safety and efficacy of factor IX gene therapy in hemophilia B","volume":"371","author":"Nathwani","year":"2014","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2024.101363_bib17","doi-asserted-by":"crossref","first-page":"23","DOI":"10.1182\/blood-2013-01-306647","article-title":"Immune responses to AAV vectors: overcoming barriers to successful gene therapy","volume":"122","author":"Mingozzi","year":"2013","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2024.101363_bib18","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1016\/j.omtm.2017.11.007","article-title":"Emerging Issues in AAV-Mediated In\u00a0Vivo Gene Therapy","volume":"8","author":"Colella","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2024.101363_bib19","doi-asserted-by":"crossref","first-page":"477","DOI":"10.1089\/hum.1998.9.4-477","article-title":"Transient Immunosuppression Allows Transgene Expression Following Readministration of Adeno-Associated Viral Vectors","volume":"9","author":"Manning","year":"1998","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib20","doi-asserted-by":"crossref","first-page":"1410","DOI":"10.1038\/mt.2012.84","article-title":"Pharmacological Modulation of Humoral Immunity in a Nonhuman Primate Model of AAV Gene Transfer for Hemophilia B","volume":"20","author":"Mingozzi","year":"2012","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib21","doi-asserted-by":"crossref","first-page":"3321","DOI":"10.1182\/blood-2006-04-017913","article-title":"Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy","volume":"108","author":"Jiang","year":"2006","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2024.101363_bib22","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-018-06621-3","article-title":"Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration","volume":"9","author":"Meliani","year":"2018","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2024.101363_bib23","doi-asserted-by":"crossref","first-page":"1870","DOI":"10.1016\/j.ymthe.2023.06.009","article-title":"Re-administration of AAV vectors by masking with host albumin: A Goldilocks hypothesis","volume":"31","author":"Wright","year":"2023","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib24","doi-asserted-by":"crossref","first-page":"2084","DOI":"10.1038\/mt.2011.108","article-title":"A 10 Patient Case Report on the Impact of Plasmapheresis Upon Neutralizing Factors Against Adeno-associated Virus (AAV) Types 1, 2, 6, and 8","volume":"19","author":"Monteilhet","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib25","doi-asserted-by":"crossref","first-page":"338","DOI":"10.1038\/mt.2013.244","article-title":"Plasmapheresis Eliminates the Negative Impact of AAV Antibodies on Microdystrophin Gene Expression Following Vascular Delivery","volume":"22","author":"Chicoine","year":"2014","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib26","doi-asserted-by":"crossref","first-page":"1831","DOI":"10.1016\/j.ymthe.2017.05.003","article-title":"Successful Repeated Hepatic Gene Delivery in Mice and Non-human Primates Achieved by Sequential Administration of AAV5 ch and AAV1","volume":"25","author":"Majowicz","year":"2017","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib27","doi-asserted-by":"crossref","first-page":"194ra92","DOI":"10.1126\/scitranslmed.3005795","article-title":"Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys","volume":"5","author":"Mingozzi","year":"2013","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtm.2024.101363_bib28","doi-asserted-by":"crossref","first-page":"1096","DOI":"10.1038\/s41591-020-0911-7","article-title":"IgG-cleaving endopeptidase enables in\u00a0vivo gene therapy in the presence of anti-AAV neutralizing antibodies","volume":"26","author":"Leborgne","year":"2020","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2024.101363_bib29","doi-asserted-by":"crossref","first-page":"825","DOI":"10.1007\/s40744-023-00546-0","article-title":"Phase 2 Dose-Finding Study in Patients with Gout Using SEL-212, a Novel PEGylated Uricase (SEL-037) Combined with Tolerogenic Nanoparticles (SEL-110)","volume":"10","author":"Kivitz","year":"2023","journal-title":"Rheumatol. Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib30","doi-asserted-by":"crossref","first-page":"272","DOI":"10.1038\/s41467-021-27945-7","article-title":"Tolerogenic nanoparticles mitigate the formation of anti-drug antibodies against pegylated uricase in patients with hyperuricemia","volume":"13","author":"Sands","year":"2022","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2024.101363_bib31","doi-asserted-by":"crossref","DOI":"10.3389\/fimmu.2021.637469","article-title":"Enhancement of the Tolerogenic Phenotype in the Liver by ImmTOR Nanoparticles","volume":"12","author":"Ilyinskii","year":"2021","journal-title":"Front. Immunol."},{"key":"10.1016\/j.omtm.2024.101363_bib32","doi-asserted-by":"crossref","first-page":"890","DOI":"10.1038\/nnano.2016.135","article-title":"Improving the efficacy and safety of biologic drugs with tolerogenic nanoparticles","volume":"11","author":"Kishimoto","year":"2016","journal-title":"Nat. Nanotechnol."},{"key":"10.1016\/j.omtm.2024.101363_bib33","doi-asserted-by":"crossref","first-page":"E156","DOI":"10.1073\/pnas.1408686111","article-title":"Polymeric synthetic nanoparticles for the induction of antigen-specific immunological tolerance","volume":"112","author":"Maldonado","year":"2015","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2024.101363_bib34","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.abd0321","article-title":"Enhancement of liver-directed transgene expression at initial and repeat doses of AAV vectors admixed with ImmTOR nanoparticles","volume":"7","author":"Ilyinskii","year":"2021","journal-title":"Sci. Adv."},{"key":"10.1016\/j.omtm.2024.101363_bib35","article-title":"Rescue of infant progressive familial intrahepatic cholestasis type 3 mice by repeated dosing of AAV gene therapy","volume":"5","author":"Weber","year":"2023","journal-title":"JHEP Rep."},{"key":"10.1016\/j.omtm.2024.101363_bib36","doi-asserted-by":"crossref","first-page":"1052","DOI":"10.1096\/fj.11-195461","article-title":"Rescue of bilirubin-induced neonatal lethality in a mouse model of Crigler-Najjar syndrome type I by AAV9-mediated gene transfer","volume":"26","author":"Bortolussi","year":"2012","journal-title":"Faseb. J."},{"key":"10.1016\/j.omtm.2024.101363_bib37","doi-asserted-by":"crossref","first-page":"24048","DOI":"10.1016\/S0021-9258(18)54390-8","article-title":"Molecular basis of multiple UDP-glucuronosyltransferase isoenzyme deficiencies in the hyperbilirubinemic rat (Gunn rat)","volume":"266","author":"Iyanagi","year":"1991","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2024.101363_bib38","first-page":"1057","article-title":"Age-dependent pattern of cerebellar susceptibility to bilirubin neurotoxicity in\u00a0vivo in mice","volume":"7","author":"Bortolussi","year":"2014","journal-title":"Dis. Model. Mech."},{"key":"10.1016\/j.omtm.2024.101363_bib39","doi-asserted-by":"crossref","first-page":"480","DOI":"10.1182\/blood-2002-09-2889","article-title":"Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway","volume":"102","author":"Davidoff","year":"2003","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2024.101363_bib40","doi-asserted-by":"crossref","first-page":"593","DOI":"10.1016\/S1525-0016(03)00234-X","article-title":"Long-term correction of bilirubin UDPglucuronyltransferase deficiency in rats by in utero lentiviral gene transfer","volume":"8","author":"Seppen","year":"2003","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib41","doi-asserted-by":"crossref","first-page":"72","DOI":"10.2174\/156652309787909508","article-title":"Towards liver-directed gene therapy for Crigler-Najjar syndrome","volume":"9","author":"Miranda","year":"2009","journal-title":"Curr. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib42","doi-asserted-by":"crossref","first-page":"3930","DOI":"10.1073\/pnas.0500930102","article-title":"Lifelong elimination of hyperbilirubinemia in the Gunn rat with a single injection of helper-dependent adenoviral vector","volume":"102","author":"Toietta","year":"2005","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2024.101363_bib43","doi-asserted-by":"crossref","first-page":"672","DOI":"10.1038\/sj.gt.3302681","article-title":"Immune response to lentiviral bilirubin UDP-glucuronosyltransferase gene transfer in fetal and neonatal rats","volume":"13","author":"Seppen","year":"2006","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib44","doi-asserted-by":"crossref","first-page":"480","DOI":"10.1182\/blood-2002-09-2889","article-title":"Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway","volume":"102","author":"Davidoff","year":"2003","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2024.101363_bib45","doi-asserted-by":"crossref","first-page":"165","DOI":"10.1038\/s41431-019-0508-0","article-title":"Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database","volume":"28","author":"Nguengang Wakap","year":"2020","journal-title":"Eur. J. Hum. Genet."},{"key":"10.1016\/j.omtm.2024.101363_bib46","doi-asserted-by":"crossref","first-page":"2519","DOI":"10.1056\/NEJMoa1708483","article-title":"AAV5-Factor VIII Gene Transfer in Severe Hemophilia A","volume":"377","author":"Rangarajan","year":"2017","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2024.101363_bib47","doi-asserted-by":"crossref","first-page":"1085","DOI":"10.1016\/j.ymthe.2006.01.014","article-title":"Adeno-associated virus vector serotypes mediate sustained correction of bilirubin UDP glucuronosyltransferase deficiency in rats","volume":"13","author":"Seppen","year":"2006","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib48","doi-asserted-by":"crossref","first-page":"649","DOI":"10.1038\/gt.2017.75","article-title":"Repeated AAV-mediated gene transfer by serotype switching enables long-lasting therapeutic levels of hUGT1A1 enzyme in a mouse model of Crigler-Najjar Syndrome Type I","volume":"24","author":"Bockor","year":"2017","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib49","article-title":"Coupling AAV-mediated promoterless gene targeting to SaCas9 nuclease to efficiently correct liver metabolic diseases","volume":"5","author":"De Caneva","year":"2019","journal-title":"JCI Insight"},{"key":"10.1016\/j.omtm.2024.101363_bib50","doi-asserted-by":"crossref","first-page":"1346","DOI":"10.15252\/emmm.201707601","article-title":"Promoterless gene targeting without nucleases rescues lethality of a Crigler-Najjar syndrome mouse model","volume":"9","author":"Porro","year":"2017","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.omtm.2024.101363_bib51","doi-asserted-by":"crossref","first-page":"763","DOI":"10.1089\/hum.2017.185","article-title":"AAV8 Gene Therapy Rescues the Newborn Phenotype of a Mouse Model of Crigler-Najjar","volume":"29","author":"Greig","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib52","doi-asserted-by":"crossref","first-page":"689","DOI":"10.1002\/jgm.1343","article-title":"Transient expression of genes delivered to newborn rat liver using recombinant adeno-associated virus 2\/8 vectors","volume":"11","author":"Flageul","year":"2009","journal-title":"J.\u00a0Gene Med."},{"key":"10.1016\/j.omtm.2024.101363_bib53","doi-asserted-by":"crossref","first-page":"306","DOI":"10.1007\/s00431-005-0055-2","article-title":"Management of hyperbilirubinemia and prevention of kernicterus in 20 patients with Crigler-Najjar disease","volume":"165","author":"Strauss","year":"2006","journal-title":"Eur. J. Pediatr."},{"key":"10.1016\/j.omtm.2024.101363_bib54","doi-asserted-by":"crossref","first-page":"1593","DOI":"10.1111\/liv.15239","article-title":"Disease burden and management of Crigler-Najjar syndrome: Report of a world registry","volume":"42","author":"Aronson","year":"2022","journal-title":"Liver Int."},{"key":"10.1016\/j.omtm.2024.101363_bib55","doi-asserted-by":"crossref","first-page":"2073","DOI":"10.1016\/j.ymthe.2020.06.001","article-title":"Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B","volume":"28","author":"George","year":"2020","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib56","doi-asserted-by":"crossref","first-page":"836","DOI":"10.1016\/j.tibtech.2022.11.006","article-title":"Evading and overcoming AAV neutralization in gene therapy","volume":"41","author":"Earley","year":"2023","journal-title":"Trends Biotechnol."},{"key":"10.1016\/j.omtm.2024.101363_bib57","first-page":"18","article-title":"A pilot study on using rapamycin-carrying synthetic vaccine particles (SVP) in conjunction with enzyme replacement therapy to induce immune tolerance in Pompe disease","volume":"13","author":"Lim","year":"2017","journal-title":"Mol. Genet. Metab. Rep."},{"key":"10.1016\/j.omtm.2024.101363_bib58","first-page":"19","article-title":"Functional assessment of T Cell responses to AAV8 empty capsids in healthy volunteers","volume":"30","author":"Schroeder","year":"2022","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib59","first-page":"336","article-title":"Effect of tolerogenic ImmTOR nanoparticles on the formation of anti-AAV8 antibodies in mice, nonhuman primates, and healthy human volunteers","volume":"30","author":"Traber","year":"2022","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib60","doi-asserted-by":"crossref","first-page":"807","DOI":"10.1089\/hum.2013.086","article-title":"Polyinosinic acid blocks adeno-associated virus macrophage endocytosis in\u00a0vitro and enhances adeno-associated virus liver-directed gene therapy in\u00a0vivo","volume":"24","author":"van Dijk","year":"2013","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib61","doi-asserted-by":"crossref","first-page":"2632","DOI":"10.1182\/bloodadvances.2019000380","article-title":"Immunoadsorption enables successful rAAV5-mediated repeated hepatic gene delivery in nonhuman primates","volume":"3","author":"Salas","year":"2019","journal-title":"Blood Adv."},{"key":"10.1016\/j.omtm.2024.101363_bib62","doi-asserted-by":"crossref","first-page":"377","DOI":"10.1038\/s41434-022-00368-9","article-title":"IgG-cleavage protein allows therapeutic AAV gene delivery in passively immunized MPS IIIA mice","volume":"30","author":"Bobo","year":"2023","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib63","doi-asserted-by":"crossref","DOI":"10.1093\/pnasnexus\/pgad394","article-title":"Readministration of high-dose adeno-associated virus gene therapy vectors enabled by ImmTOR nanoparticles combined with B cell-targeted agents","volume":"2","author":"Ilyinskii","year":"2023","journal-title":"PNAS Nexus"},{"key":"10.1016\/j.omtm.2024.101363_bib64","doi-asserted-by":"crossref","first-page":"244","DOI":"10.1016\/j.lfs.2015.10.025","article-title":"Men and mice: Relating their ages","volume":"152","author":"Dutta","year":"2016","journal-title":"Life Sci."},{"key":"10.1016\/j.omtm.2024.101363_bib65","doi-asserted-by":"crossref","first-page":"279","DOI":"10.1016\/j.omtm.2021.06.015","article-title":"ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia","volume":"22","author":"Ilyinskii","year":"2021","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2024.101363_bib66","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1089\/hum.1997.8.1-27","article-title":"Transplantation of Gunn Rats with Autologous Fibroblasts Expressing Bilirubin UDP-Glucuronosyltransferase: Correction of Genetic Deficiency and Tumor Formation","volume":"8","author":"Seppen","year":"1997","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib67","doi-asserted-by":"crossref","first-page":"168","DOI":"10.1038\/gt.2013.69","article-title":"In the rat liver, Adenoviral gene transfer efficiency is comparable to AAV","volume":"21","author":"Montenegro-Miranda","year":"2014","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2024.101363_bib68","doi-asserted-by":"crossref","first-page":"153","DOI":"10.1016\/j.jhep.2019.03.021","article-title":"Liver-directed gene therapy results in long-term correction of progressive familial intrahepatic cholestasis type 3 in mice","volume":"71","author":"Aronson","year":"2019","journal-title":"J.\u00a0Hepatol."},{"key":"10.1016\/j.omtm.2024.101363_bib69","doi-asserted-by":"crossref","first-page":"45","DOI":"10.1089\/hgtb.2015.037","article-title":"Determination of Anti-Adeno-Associated Virus Vector Neutralizing Antibody Titer with an In\u00a0Vitro Reporter System","volume":"26","author":"Meliani","year":"2015","journal-title":"Hum. Gene Ther. Methods"}],"container-title":["Molecular Therapy - Methods & Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050124001797?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050124001797?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,1,23]],"date-time":"2025-01-23T21:06:28Z","timestamp":1737666388000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050124001797"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,12]]},"references-count":69,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2024,12]]}},"alternative-id":["S2329050124001797"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2024.101363","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2024,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2024.101363","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2024 The Authors. Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}],"article-number":"101363"}}