{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,12]],"date-time":"2026-09-12T05:17:03Z","timestamp":1789190223533,"version":"build-2803163510"},"reference-count":39,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2023,6,1]],"date-time":"2023-06-01T00:00:00Z","timestamp":1685577600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2023,6,1]],"date-time":"2023-06-01T00:00:00Z","timestamp":1685577600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2023,2,13]],"date-time":"2023-02-13T00:00:00Z","timestamp":1676246400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy - Methods & Clinical Development"],"published-print":{"date-parts":[[2023,6]]},"DOI":"10.1016\/j.omtm.2023.02.007","type":"journal-article","created":{"date-parts":[[2023,2,15]],"date-time":"2023-02-15T08:03:47Z","timestamp":1676448227000},"page":"32-39","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":15,"special_numbering":"C","title":["Lipid nanoparticle-encapsulated mRNA therapy corrects serum total bilirubin level in Crigler-Najjar syndrome mouse model"],"prefix":"10.1016","volume":"29","author":[{"given":"Jenny A.","family":"Greig","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joanna K.","family":"Chorazeczewski","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1277-0128","authenticated-orcid":false,"given":"Vivek","family":"Chowdhary","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melanie K.","family":"Smith","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthew","family":"Jennis","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James C.","family":"Tarrant","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elizabeth L.","family":"Buza","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kimberly","family":"Coughlan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paolo G.V.","family":"Martini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9630-3131","authenticated-orcid":false,"given":"James M.","family":"Wilson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2023.02.007_bib1","first-page":"169","article-title":"Congenital familial nonhemolytic jaundice with kernicterus","volume":"10","author":"Crigler","year":"1952","journal-title":"Pediatrics"},{"key":"10.1016\/j.omtm.2023.02.007_bib2","first-page":"259","article-title":"Congenital familial nonhemolytic jaundice with kernicterus; a new clinical entity","volume":"83","author":"Crigler","year":"1952","journal-title":"AMA. Am. J. Dis. Child."},{"key":"10.1016\/j.omtm.2023.02.007_bib3","doi-asserted-by":"crossref","first-page":"1043","DOI":"10.1016\/S0021-9258(17)35280-8","article-title":"Cloning of two human liver bilirubin UDP-glucuronosyltransferase cDNAs with expression in COS-1 cells","volume":"266","author":"Ritter","year":"1991","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2023.02.007_bib4","doi-asserted-by":"crossref","first-page":"941","DOI":"10.1002\/hep.1840150531","article-title":"Sequence of exons and the flanking regions of human bilirubin-UDP-glucuronosyltransferase gene complex and identification of a genetic mutation in a patient with Crigler-Najjar syndrome, type I","volume":"15","author":"Bosma","year":"1992","journal-title":"Hepatology"},{"key":"10.1016\/j.omtm.2023.02.007_bib5","doi-asserted-by":"crossref","first-page":"150","DOI":"10.1172\/JCI115829","article-title":"Identification of a genetic alteration in the code for bilirubin UDP-glucuronosyltransferase in the UGT1 gene complex of a Crigler-Najjar type I patient","volume":"90","author":"Ritter","year":"1992","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2023.02.007_bib6","doi-asserted-by":"crossref","first-page":"395","DOI":"10.1016\/0002-9343(69)90224-1","article-title":"Chronic nonhemolytic unconjugated hyperbilirubinemia with glucuronyl transferase deficiency. Clinical, biochemical, pharmacologic and genetic evidence for heterogeneity","volume":"47","author":"Arias","year":"1969","journal-title":"Am. J. Med."},{"key":"10.1016\/j.omtm.2023.02.007_bib7","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.2023.02.007_bib8","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.2023.02.007_bib9","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.2023.02.007_bib10","doi-asserted-by":"crossref","first-page":"191","DOI":"10.1016\/j.omtm.2018.10.012","article-title":"AAV8 gene therapy for Crigler-Najjar syndrome in macaques elicited transgene T\u00a0cell responses that are resident to the liver","volume":"11","author":"Greig","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2023.02.007_bib11","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.2023.02.007_bib12","doi-asserted-by":"crossref","first-page":"237","DOI":"10.1016\/j.omtm.2018.07.008","article-title":"Determining the minimally effective dose of a clinical candidate AAV vector in a mouse model of Crigler-Najjar syndrome","volume":"10","author":"Greig","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2023.02.007_bib13","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":"Bo\u010dkor","year":"2017","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2023.02.007_bib14","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.2023.02.007_bib15","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.2023.02.007_bib16","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.2023.02.007_bib17","doi-asserted-by":"crossref","first-page":"16049","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.2023.02.007_bib18","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.2023.02.007_bib19","doi-asserted-by":"crossref","first-page":"404","DOI":"10.1002\/psp4.12301","article-title":"Quantitative systems pharmacology model of hUGT1A1-modRNA encoding for the UGT1A1 enzyme to treat Crigler-Najjar syndrome type 1","volume":"7","author":"Apgar","year":"2018","journal-title":"CPT Pharmacometrics Syst. Pharmacol."},{"key":"10.1016\/j.omtm.2023.02.007_bib20","doi-asserted-by":"crossref","first-page":"1467","DOI":"10.1016\/j.ymthe.2017.03.013","article-title":"Lipid nanoparticle systems for enabling gene therapies","volume":"25","author":"Cullis","year":"2017","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2023.02.007_bib21","doi-asserted-by":"crossref","first-page":"7300","DOI":"10.1021\/acs.nanolett.5b02497","article-title":"Optimization of lipid nanoparticle formulations for mRNA delivery in\u00a0vivo with fractional factorial and definitive screening designs","volume":"15","author":"Kauffman","year":"2015","journal-title":"Nano Lett."},{"key":"10.1016\/j.omtm.2023.02.007_bib22","doi-asserted-by":"crossref","first-page":"78","DOI":"10.1016\/j.biomaterials.2016.09.006","article-title":"Efficacy and immunogenicity of unmodified and pseudouridine-modified mRNA delivered systemically with lipid nanoparticles in\u00a0vivo","volume":"109","author":"Kauffman","year":"2016","journal-title":"Biomaterials"},{"key":"10.1016\/j.omtm.2023.02.007_bib23","doi-asserted-by":"crossref","first-page":"345","DOI":"10.1016\/j.jconrel.2015.08.007","article-title":"Expression kinetics of nucleoside-modified mRNA delivered in lipid nanoparticles to mice by various routes","volume":"217","author":"Pardi","year":"2015","journal-title":"J.\u00a0Contr. Release"},{"key":"10.1016\/j.omtm.2023.02.007_bib24","doi-asserted-by":"crossref","first-page":"14630","DOI":"10.1038\/ncomms14630","article-title":"Administration of nucleoside-modified mRNA encoding broadly neutralizing antibody protects humanized mice from HIV-1 challenge","volume":"8","author":"Pardi","year":"2017","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2023.02.007_bib25","doi-asserted-by":"crossref","first-page":"E1941","DOI":"10.1073\/pnas.1619653114","article-title":"Systemic delivery of factor IX messenger RNA for protein replacement therapy","volume":"114","author":"Ramaswamy","year":"2017","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2023.02.007_bib26","doi-asserted-by":"crossref","first-page":"21302","DOI":"10.1016\/S0021-9258(19)30079-1","article-title":"The 3-methylcholanthrene-inducible UDP-glucuronosyltransferase deficiency in the hyperbilirubinemic rat (Gunn rat) is caused by a -1 frameshift mutation","volume":"264","author":"Iyanagi","year":"1989","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2023.02.007_bib27","first-page":"230","article-title":"Hereditary acholuric jaundice in the rat","volume":"50","author":"Gunn","year":"1944","journal-title":"Can. Med. Assoc. J."},{"key":"10.1016\/j.omtm.2023.02.007_bib28","doi-asserted-by":"crossref","first-page":"7901","DOI":"10.1074\/jbc.M709244200","article-title":"Disruption of the ugt1 locus in mice resembles human Crigler-Najjar type I disease","volume":"283","author":"Nguyen","year":"2008","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2023.02.007_bib29","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.2023.02.007_bib30","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.2023.02.007_bib31","doi-asserted-by":"crossref","first-page":"519","DOI":"10.1016\/j.ebiom.2019.07.003","article-title":"Long-term efficacy and safety of mRNA therapy in two murine models of methylmalonic acidemia","volume":"45","author":"An","year":"2019","journal-title":"EBioMedicine"},{"key":"10.1016\/j.omtm.2023.02.007_bib32","doi-asserted-by":"crossref","first-page":"3548","DOI":"10.1016\/j.celrep.2017.11.081","article-title":"Systemic messenger RNA therapy as a treatment for methylmalonic acidemia","volume":"21","author":"An","year":"2017","journal-title":"Cell Rep."},{"key":"10.1016\/j.omtm.2023.02.007_bib33","doi-asserted-by":"crossref","first-page":"S89","DOI":"10.1007\/PL00014330","article-title":"Diagnosis and management of Crigler-Najjar syndrome","volume":"158","author":"Jansen","year":"1999","journal-title":"Eur. J. Pediatr."},{"key":"10.1016\/j.omtm.2023.02.007_bib34","doi-asserted-by":"crossref","first-page":"1227","DOI":"10.1016\/S0022-3468(03)00273-2","article-title":"Treatment of Crigler-Najjar type 1 disease: relevance of early liver transplantation","volume":"38","author":"Schauer","year":"2003","journal-title":"J.\u00a0Pediatr. Surg."},{"key":"10.1016\/j.omtm.2023.02.007_bib35","doi-asserted-by":"crossref","first-page":"311","DOI":"10.1002\/hep.510240205","article-title":"Current therapy for Crigler-Najjar syndrome type 1: report of a world registry","volume":"24","author":"van der Veere","year":"1996","journal-title":"Hepatology"},{"key":"10.1016\/j.omtm.2023.02.007_bib36","doi-asserted-by":"crossref","first-page":"522","DOI":"10.1177\/1093526617697059","article-title":"Liver fibrosis associated with Crigler-Najjar syndrome in a compound heterozygote: a case report","volume":"20","author":"Fata","year":"2017","journal-title":"Pediatr. Dev. Pathol."},{"key":"10.1016\/j.omtm.2023.02.007_bib37","doi-asserted-by":"crossref","first-page":"eaaz6893","DOI":"10.1126\/sciadv.aaz6893","article-title":"Impact of mRNA chemistry and manufacturing process on innate immune activation","volume":"6","author":"Nelson","year":"2020","journal-title":"Sci. Adv."},{"key":"10.1016\/j.omtm.2023.02.007_bib38","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.omtn.2019.01.013","article-title":"Optimization of lipid nanoparticles for intramuscular administration of mRNA vaccines","volume":"15","author":"Hassett","year":"2019","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtm.2023.02.007_bib39","doi-asserted-by":"crossref","first-page":"671","DOI":"10.1038\/nmeth.2089","article-title":"NIH Image to ImageJ: 25 years of image analysis","volume":"9","author":"Schneider","year":"2012","journal-title":"Nat. Methods"}],"container-title":["Molecular Therapy - Methods & Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050123000177?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050123000177?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2024,5,17]],"date-time":"2024-05-17T13:36:40Z","timestamp":1715953000000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050123000177"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,6]]},"references-count":39,"alternative-id":["S2329050123000177"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2023.02.007","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2023,6]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Lipid nanoparticle-encapsulated mRNA therapy corrects serum total bilirubin level in Crigler-Najjar syndrome mouse model","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2023.02.007","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"}]}}