{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,18]],"date-time":"2026-09-18T15:14:32Z","timestamp":1789744472285,"version":"4.0.1"},"reference-count":38,"publisher":"Elsevier BV","issue":"6","license":[{"start":{"date-parts":[[2006,6,1]],"date-time":"2006-06-01T00:00:00Z","timestamp":1149120000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2016,10,7]],"date-time":"2016-10-07T00:00:00Z","timestamp":1475798400000},"content-version":"vor","delay-in-days":3781,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2006,6]]},"DOI":"10.1016\/j.ymthe.2006.01.014","type":"journal-article","created":{"date-parts":[[2006,4,5]],"date-time":"2006-04-05T14:48:41Z","timestamp":1144248521000},"page":"1085-1092","source":"Crossref","is-referenced-by-count":78,"title":["Adeno-associated Virus Vector Serotypes Mediate Sustained Correction of Bilirubin UDP Glucuronosyltransferase Deficiency in Rats"],"prefix":"10.1016","volume":"13","author":[{"given":"Jurgen","family":"Seppen","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Conny","family":"Bakker","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Berry","family":"de Jong","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cindy","family":"Kunne","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karin","family":"van den Oever","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristin","family":"Vandenberghe","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rudi","family":"de Waart","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jaap","family":"Twisk","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Piter","family":"Bosma","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymthe.2006.01.014_bib1","doi-asserted-by":"crossref","first-page":"1715","DOI":"10.1016\/S0022-2275(20)39768-6","article-title":"Structure and binding of unconjugated bilirubin: relevance for physiological and pathophysiological function","volume":"35","author":"Ostrow","year":"1994","journal-title":"J. Lipid Res."},{"key":"10.1016\/j.ymthe.2006.01.014_bib2","doi-asserted-by":"crossref","first-page":"581","DOI":"10.1146\/annurev.pharmtox.40.1.581","article-title":"Human UDP-glucuronosyltransferases: metabolism, expression, and disease","volume":"40","author":"Tukey","year":"2000","journal-title":"Annu. Rev. Pharmacol. Toxicol."},{"issue":"Suppl. 2","key":"10.1016\/j.ymthe.2006.01.014_bib3","doi-asserted-by":"crossref","first-page":"S89","DOI":"10.1007\/PL00014330","article-title":"Diagnosis and management of Crigler\u2013Najjar syndrome","volume":"158","author":"Jansen","year":"1999","journal-title":"Eur. J. Pediatr."},{"key":"10.1016\/j.ymthe.2006.01.014_bib4","doi-asserted-by":"crossref","first-page":"1277","DOI":"10.1053\/jhep.2002.33432","article-title":"Mechanisms of bilirubin neurotoxicity","volume":"35","author":"Ostrow","year":"2002","journal-title":"Hepatology"},{"key":"10.1016\/j.ymthe.2006.01.014_bib5","first-page":"149","article-title":"Gunn rat: a model for inherited deficiency of bilirubin glucuronidation","volume":"37","author":"Chowdhury","year":"1993","journal-title":"Adv. Vet. Sci. Comp. Med."},{"key":"10.1016\/j.ymthe.2006.01.014_bib6","doi-asserted-by":"crossref","first-page":"1279","DOI":"10.1089\/hum.2004.15.1279","article-title":"Nonviral gene transfer into liver and muscle for treatment of hyperbilirubinemia in the Gunn rat","volume":"15","author":"Danko","year":"2004","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2006.01.014_bib7","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.ymthe.2006.01.014_bib8","doi-asserted-by":"crossref","first-page":"1348","DOI":"10.1053\/gast.2000.19577","article-title":"A replication-deficient rSV40 mediates liver-directed gene transfer and a long-term amelioration of jaundice in Gunn rats","volume":"119","author":"Sauter","year":"2000","journal-title":"Gastroenterology"},{"key":"10.1016\/j.ymthe.2006.01.014_bib9","first-page":"381","article-title":"Complete correction of hyperbilirubinemia in the Gunn rat model of Crigler\u2013Najjar syndrome type I following transient in vivo adenovirus-mediated expression of human bilirubin UDP-glucuronosyltransferase","volume":"3","author":"Askari","year":"1996","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2006.01.014_bib10","doi-asserted-by":"crossref","first-page":"26536","DOI":"10.1074\/jbc.271.43.26536","article-title":"Long term correction of bilirubin\u2013UDP-glucuronosyltransferase deficiency in Gunn rats by administration of a recombinant adenovirus during the neonatal period","volume":"271","author":"Takahashi","year":"1996","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.ymthe.2006.01.014_bib11","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.ymthe.2006.01.014_bib12","doi-asserted-by":"crossref","first-page":"431","DOI":"10.1002\/hep.20794","article-title":"Successful gene therapy of the Gunn rat by in vivo neonatal hepatic gene transfer using murine oncoretroviral vectors","volume":"42","author":"Bellodi-Privato","year":"2005","journal-title":"Hepatology"},{"key":"10.1016\/j.ymthe.2006.01.014_bib13","doi-asserted-by":"crossref","first-page":"78","DOI":"10.1002\/lt.500040111","article-title":"Long-term reduction of serum bilirubin levels in Gunn rats by retroviral gene transfer in vivo","volume":"4","author":"Tada","year":"1998","journal-title":"Liver Transplant. Surg."},{"key":"10.1016\/j.ymthe.2006.01.014_bib14","doi-asserted-by":"crossref","first-page":"593","DOI":"10.1016\/S1525-0016(03)00234-X","article-title":"Elferink, 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.ymthe.2006.01.014_bib15","doi-asserted-by":"crossref","first-page":"852","DOI":"10.1016\/j.ymthe.2005.06.482","article-title":"Therapeutic lentivirus-mediated neonatal in vivo gene therapy in hyperbilirubinemic Gunn rats","volume":"12","author":"Nguyen","year":"2005","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2006.01.014_bib16","doi-asserted-by":"crossref","first-page":"541","DOI":"10.1089\/hum.2005.16.541","article-title":"Adeno-associated virus vectors in clinical trials","volume":"16","author":"Carter","year":"2005","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2006.01.014_bib17","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1038\/73464","article-title":"Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector","volume":"24","author":"Kay","year":"2000","journal-title":"Nat. Genet."},{"key":"10.1016\/j.ymthe.2006.01.014_bib18","doi-asserted-by":"crossref","first-page":"11343","DOI":"10.1128\/JVI.76.22.11343-11349.2002","article-title":"A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction","volume":"76","author":"Nakai","year":"2002","journal-title":"J. Virol."},{"key":"10.1016\/j.ymthe.2006.01.014_bib19","doi-asserted-by":"crossref","first-page":"3994","DOI":"10.1128\/JVI.73.5.3994-4003.1999","article-title":"Gene therapy vectors based on adeno-associated virus type 1","volume":"73","author":"Xiao","year":"1999","journal-title":"J. Virol."},{"key":"10.1016\/j.ymthe.2006.01.014_bib20","doi-asserted-by":"crossref","first-page":"309","DOI":"10.1128\/JVI.72.1.309-319.1998","article-title":"Infectious clones and vectors derived from adeno-associated virus (AAV) serotypes other than AAV type 2","volume":"72","author":"Rutledge","year":"1998","journal-title":"J. Virol."},{"key":"10.1016\/j.ymthe.2006.01.014_bib21","doi-asserted-by":"crossref","first-page":"11854","DOI":"10.1073\/pnas.182412299","article-title":"Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy","volume":"99","author":"Gao","year":"2002","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2006.01.014_bib22","doi-asserted-by":"crossref","first-page":"875","DOI":"10.1016\/j.ymthe.2004.12.022","article-title":"Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models","volume":"11","author":"Davidoff","year":"2005","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2006.01.014_bib23","doi-asserted-by":"crossref","first-page":"2412","DOI":"10.1182\/blood-2003-02-0495","article-title":"Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy","volume":"102","author":"Grimm","year":"2003","journal-title":"Blood"},{"key":"10.1016\/j.ymthe.2006.01.014_bib24","doi-asserted-by":"crossref","first-page":"214","DOI":"10.1128\/JVI.79.1.214-224.2005","article-title":"Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice","volume":"79","author":"Nakai","year":"2005","journal-title":"J. Virol."},{"key":"10.1016\/j.ymthe.2006.01.014_bib25","doi-asserted-by":"crossref","first-page":"791","DOI":"10.1128\/JVI.76.2.791-801.2002","article-title":"Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity","volume":"76","author":"Rabinowitz","year":"2002","journal-title":"J. Virol."},{"key":"10.1016\/j.ymthe.2006.01.014_bib26","doi-asserted-by":"crossref","first-page":"2295","DOI":"10.1089\/10430349950016942","article-title":"Enhanced expression of transgenes from adeno-associated virus vectors with the woodchuck hepatitis virus posttranscriptional regulatory element: implications for gene therapy","volume":"10","author":"Loeb","year":"1999","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2006.01.014_bib27","doi-asserted-by":"crossref","first-page":"268","DOI":"10.1101\/gad.1.3.268","article-title":"An albumin enhancer located 10 kb upstream functions along with its promoter to direct efficient, liver-specific expression in transgenic mice","volume":"1","author":"Pinkert","year":"1987","journal-title":"Genes Dev."},{"key":"10.1016\/j.ymthe.2006.01.014_bib28","doi-asserted-by":"crossref","DOI":"10.1038\/sj.gt.3302681","article-title":"Immune response to lentiviral bilirubin UDP-glucuronosyltransferase gene transfer in fetal and neonatal rats","author":"Seppen","year":"2006","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2006.01.014_bib29","doi-asserted-by":"crossref","first-page":"299","DOI":"10.1089\/hum.2005.16.299","article-title":"Modified infusion procedures affect recombinant adeno-associated virus vector type 2 transduction in the liver","volume":"16","author":"Ohashi","year":"2005","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2006.01.014_bib30","doi-asserted-by":"crossref","first-page":"1949","DOI":"10.1128\/JVI.73.3.1949-1955.1999","article-title":"Structure of adeno-associated virus vector DNA following transduction of the skeletal muscle","volume":"73","author":"Vincent-Lacaze","year":"1999","journal-title":"J. Virol."},{"key":"10.1016\/j.ymthe.2006.01.014_bib31","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.ymthe.2006.01.014_bib32","doi-asserted-by":"crossref","first-page":"299","DOI":"10.1016\/j.ymthe.2005.03.020","article-title":"No evidence for tumorigenesis of AAV vectors in a large-scale study in mice","volume":"12","author":"Bell","year":"2005","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2006.01.014_bib33","doi-asserted-by":"crossref","first-page":"906","DOI":"10.1089\/hum.2004.15.906","article-title":"Long-term correction of murine lipoprotein lipase deficiency with AAV1-mediated gene transfer of the naturally occurring LPL(S447X) beneficial mutation","volume":"15","author":"Ross","year":"2004","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2006.01.014_bib34","doi-asserted-by":"crossref","first-page":"2727","DOI":"10.1089\/hum.1998.9.18-2727","article-title":"Long-term genetic modification of rhesus monkey hematopoietic cells following transplantation of adenoassociated virus vector-transduced CD34+ cells","volume":"9","author":"Schimmenti","year":"1998","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2006.01.014_bib35","doi-asserted-by":"crossref","first-page":"839","DOI":"10.1016\/S1525-0016(03)00095-9","article-title":"Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6","volume":"7","author":"Grimm","year":"2003","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2006.01.014_bib36","doi-asserted-by":"crossref","first-page":"1885","DOI":"10.1089\/10430349950017563","article-title":"Purification of recombinant adeno-associated virus by iodixanol gradient ultracentrifugation allows rapid and reproducible preparation of vector stocks for gene transfer in the nervous system","volume":"10","author":"Hermens","year":"1999","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2006.01.014_bib37","series-title":"Molecular Cloning: A Laboratory Manual","author":"Maniatis","year":"1989"},{"key":"10.1016\/j.ymthe.2006.01.014_bib38","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1007\/s004180100297","article-title":"Optimisation of oil red O staining permits combination with immunofluorescence and automated quantification of lipids","volume":"116","author":"Koopman","year":"2001","journal-title":"Histochem. Cell Biol."}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S152500160600058X?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S152500160600058X?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2021,7,25]],"date-time":"2021-07-25T10:30:54Z","timestamp":1627209054000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S152500160600058X"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2006,6]]},"references-count":38,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2006,6]]}},"alternative-id":["S152500160600058X"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2006.01.014","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2006,6]]}}}