{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T21:55:42Z","timestamp":1790805342622,"version":"4.1.0"},"reference-count":65,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2016,1,1]],"date-time":"2016-01-01T00:00:00Z","timestamp":1451606400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2017,2,18]],"date-time":"2017-02-18T00:00:00Z","timestamp":1487376000000},"content-version":"vor","delay-in-days":414,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Molecular Therapy - Methods &amp; Clinical Development"],"published-print":{"date-parts":[[2016]]},"DOI":"10.1038\/mtm.2016.49","type":"journal-article","created":{"date-parts":[[2016,7,20]],"date-time":"2016-07-20T11:11:12Z","timestamp":1469013072000},"page":"16049","source":"Crossref","is-referenced-by-count":68,"special_numbering":"C","title":["A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome"],"prefix":"10.1016","volume":"3","author":[{"given":"Giuseppe","family":"Ronzitti","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giulia","family":"Bortolussi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Remco","family":"van Dijk","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fanny","family":"Collaud","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Severine","family":"Charles","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christian","family":"Leborgne","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrice","family":"Vidal","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Samia","family":"Martin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bernard","family":"Gjata","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marcelo Simon","family":"Sola","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laetitia","family":"van Wittenberghe","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alban","family":"Vignaud","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Philippe","family":"Veron","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Piter J","family":"Bosma","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andres F","family":"Muro","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Federico","family":"Mingozzi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1038\/mtm.2016.49_bib1","first-page":"169","article-title":"Congenital familial nonhemolytic jaundice with kernicterus","volume":"10","author":"Crigler","year":"1952","journal-title":"Pediatr"},{"key":"10.1038\/mtm.2016.49_bib2","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":"Hepatol"},{"key":"10.1038\/mtm.2016.49_bib3","first-page":"3063","article-title":"Hereditary jaundice and disorders of bilirubin metabolism","volume":"vol. 2","author":"Chowdhury","year":"2001"},{"key":"10.1038\/mtm.2016.49_bib4","doi-asserted-by":"crossref","first-page":"1625","DOI":"10.1053\/j.gastro.2014.03.047","article-title":"Inherited disorders of bilirubin transport and conjugation: new insights into molecular mechanisms and consequences","volume":"146","author":"Erlinger","year":"2014","journal-title":"Gastroenterol"},{"key":"10.1038\/mtm.2016.49_bib5","doi-asserted-by":"crossref","first-page":"107","DOI":"10.1016\/S0168-8278(02)00359-8","article-title":"Inherited disorders of bilirubin metabolism","volume":"38","author":"Bosma","year":"2003","journal-title":"J Hepatol"},{"key":"10.1038\/mtm.2016.49_bib6","doi-asserted-by":"crossref","first-page":"297","DOI":"10.1002\/1098-1004(200010)16:4<297::AID-HUMU2>3.0.CO;2-Z","article-title":"Genetic lesions of bilirubin uridine-diphosphoglucuronate glucuronosyltransferase (UGT1A1) causing Crigler-Najjar and Gilbert syndromes: correlation of genotype to phenotype","volume":"16","author":"Kadakol","year":"2000","journal-title":"Hum Mutat"},{"key":"10.1038\/mtm.2016.49_bib7","doi-asserted-by":"crossref","first-page":"1146","DOI":"10.1016\/j.jpeds.2012.11.042","article-title":"UGT1A1 genetic analysis as a diagnostic aid for individuals with unconjugated hyperbilirubinemia","volume":"162","author":"Skierka","year":"2013","journal-title":"J Pediatr"},{"issue":"4 Pt 2","key":"10.1038\/mtm.2016.49_bib8","doi-asserted-by":"crossref","first-page":"768","DOI":"10.1542\/peds.89.4.768","article-title":"Cerebellar symptoms as the presenting manifestations of bilirubin encephalopathy in children with Crigler-Najjar type I disease","volume":"89","author":"Labrune","year":"1992","journal-title":"Pediatr"},{"key":"10.1038\/mtm.2016.49_bib9","doi-asserted-by":"crossref","first-page":"707","DOI":"10.1172\/JCI106541","article-title":"Photocatabolism of labeled bilirubin in the congenitally jaundiced (Gunn) rat","volume":"50","author":"Ostrow","year":"1971","journal-title":"J Clin Invest"},{"key":"10.1038\/mtm.2016.49_bib10","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":"10.1038\/mtm.2016.49_bib11","doi-asserted-by":"crossref","first-page":"33","DOI":"10.1007\/8904_2011_40","article-title":"Successful plasmapheresis for acute and severe unconjugated hyperbilirubinemia in a child with crigler najjar type I syndrome","volume":"2","author":"Sellier","year":"2012","journal-title":"JIMD Rep"},{"key":"10.1038\/mtm.2016.49_bib12","doi-asserted-by":"crossref","first-page":"961","DOI":"10.2169\/internalmedicine.39.961","article-title":"Bilirubin adsorption therapy and subsequent liver transplantation cured severe bilirubin encephalopathy in a long-term survival patient with Crigler-Najjar disease type I","volume":"39","author":"Kaneko","year":"2000","journal-title":"Intern Med"},{"key":"10.1038\/mtm.2016.49_bib13","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 Hepatol"},{"key":"10.1038\/mtm.2016.49_bib14","doi-asserted-by":"crossref","first-page":"2290","DOI":"10.1016\/j.transproceed.2014.07.039","article-title":"Predictability and survival in liver replantransplantation: monocentric experience","volume":"46","author":"Immordino","year":"2014","journal-title":"Transplantation proceedings"},{"key":"10.1038\/mtm.2016.49_bib15","doi-asserted-by":"crossref","first-page":"347","DOI":"10.1016\/j.jceh.2014.07.004","article-title":"Infections after orthotopic liver transplantation","volume":"4","author":"Pedersen","year":"2014","journal-title":"J Clin Exp Hepatol"},{"key":"10.1038\/mtm.2016.49_bib16","doi-asserted-by":"crossref","first-page":"342","DOI":"10.1038\/nm1358","article-title":"Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response","volume":"12","author":"Manno","year":"2006","journal-title":"Nat Med"},{"key":"10.1038\/mtm.2016.49_bib17","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 Engl J Med"},{"key":"10.1038\/mtm.2016.49_bib18","doi-asserted-by":"crossref","first-page":"2357","DOI":"10.1056\/NEJMoa1108046","article-title":"Adenovirus-associated virus vector-mediated gene transfer in hemophilia B","volume":"365","author":"Nathwani","year":"2011","journal-title":"N Engl J Med"},{"key":"10.1038\/mtm.2016.49_bib19","series-title":"Parvoviridae: The Viruses and their Replication","author":"Muzyczka","year":"2001"},{"key":"10.1038\/mtm.2016.49_bib20","doi-asserted-by":"crossref","first-page":"3038","DOI":"10.1182\/blood-2011-09-382317","article-title":"Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer","volume":"119","author":"Buchlis","year":"2012","journal-title":"Blood"},{"key":"10.1038\/mtm.2016.49_bib21","doi-asserted-by":"crossref","first-page":"341","DOI":"10.1038\/nrg2988","article-title":"Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges","volume":"12","author":"Mingozzi","year":"2011","journal-title":"Nat Rev Genet"},{"key":"10.1038\/mtm.2016.49_bib22","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":"10.1038\/mtm.2016.49_bib23","doi-asserted-by":"crossref","first-page":"1082","DOI":"10.1089\/hum.2012.018","article-title":"Sustained reduction of hyperbilirubinemia in Gunn rats after adeno-associated virus-mediated gene transfer of bilirubin UDP-glucuronosyltransferase isozyme 1A1 to skeletal muscle","volume":"23","author":"Pastore","year":"2012","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mtm.2016.49_bib24","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":"10.1038\/mtm.2016.49_bib25","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":"10.1038\/mtm.2016.49_bib26","doi-asserted-by":"crossref","first-page":"433","DOI":"10.1097\/MPG.0000000000000703","article-title":"Adeno-associated virus vector-based gene therapy for monogenetic metabolic diseases of the liver","volume":"60","author":"Junge","year":"2015","journal-title":"J Pediatr Gastroenterol Nutr"},{"key":"10.1038\/mtm.2016.49_bib27","doi-asserted-by":"crossref","first-page":"595","DOI":"10.1016\/j.jhep.2013.04.004","article-title":"Monogenic diseases that can be cured by liver transplantation","volume":"59","author":"Fagiuoli","year":"2013","journal-title":"J Hepatol"},{"key":"10.1038\/mtm.2016.49_bib28","doi-asserted-by":"crossref","first-page":"1422","DOI":"10.1056\/NEJM199805143382004","article-title":"Treatment of the Crigler-Najjar syndrome type I with hepatocyte transplantation","volume":"338","author":"Fox","year":"1998","journal-title":"N Engl J Med"},{"key":"10.1038\/mtm.2016.49_bib29","doi-asserted-by":"crossref","first-page":"52","DOI":"10.1002\/humu.21133","article-title":"Crigler-Najjar syndrome in The Netherlands: identification of four novel UGT1A1 alleles, genotype-phenotype correlation, and functional analysis of 10 missense mutants","volume":"31","author":"Sneitz","year":"2010","journal-title":"Hum Mutat"},{"key":"10.1038\/mtm.2016.49_bib30","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 Biol Chem"},{"key":"10.1038\/mtm.2016.49_bib31","doi-asserted-by":"crossref","first-page":"2653","DOI":"10.1182\/blood-2005-10-4035","article-title":"Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver","volume":"107","author":"Nathwani","year":"2006","journal-title":"Blood"},{"key":"10.1038\/mtm.2016.49_bib32","doi-asserted-by":"crossref","first-page":"1281","DOI":"10.1093\/nar\/15.3.1281","article-title":"The codon Adaptation Index\u2013a measure of directional synonymous codon usage bias, and its potential applications","volume":"15","author":"Sharp","year":"1987","journal-title":"Nucleic Acids Res"},{"key":"10.1038\/mtm.2016.49_bib33","doi-asserted-by":"crossref","first-page":"10770","DOI":"10.1073\/pnas.0902269106","article-title":"Cellular immune response to cryptic epitopes during therapeutic gene transfer","volume":"106","author":"Li","year":"2009","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mtm.2016.49_bib34","doi-asserted-by":"crossref","first-page":"1891","DOI":"10.1089\/hum.1997.8.16-1891","article-title":"Efficient and stable adeno-associated virus-mediated transduction in the skeletal muscle of adult immunocompetent mice","volume":"8","author":"Snyder","year":"1997","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mtm.2016.49_bib35","doi-asserted-by":"crossref","first-page":"522","DOI":"10.1006\/mthe.2000.0075","article-title":"Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro","volume":"1","author":"Miao","year":"2000","journal-title":"Mol Ther"},{"key":"10.1038\/mtm.2016.49_bib36","doi-asserted-by":"crossref","first-page":"1057","DOI":"10.1242\/dmm.016535","article-title":"Age-dependent pattern of cerebellar susceptibility to bilirubin neurotoxicity in vivo in mice","volume":"7","author":"Bortolussi","year":"2014","journal-title":"Dis Model Mech"},{"key":"10.1038\/mtm.2016.49_bib37","doi-asserted-by":"crossref","first-page":"151","DOI":"10.3727\/000000005783983250","article-title":"Isolated hepatocyte transplantation for Crigler-Najjar syndrome type 1","volume":"14","author":"Ambrosino","year":"2005","journal-title":"Cell Transplant"},{"issue":"6 Pt 1","key":"10.1038\/mtm.2016.49_bib38","doi-asserted-by":"crossref","first-page":"1262","DOI":"10.1542\/peds.111.6.1262","article-title":"Isolated hepatocyte transplantation in an infant with a severe urea cycle disorder","volume":"111","author":"Horslen","year":"2003","journal-title":"Pediatr"},{"key":"10.1038\/mtm.2016.49_bib39","first-page":"O-027","article-title":"Phase I\/II multicenter trial of liver derived mesenchymal stem cells (HepastemR) for the treatment of urea cycle disorder and Crigler Najjar syndrome interim analysis at 6 months post infusion","volume":"37","author":"Dobbelaere","year":"2014","journal-title":"J Inherited Metab Dis"},{"key":"10.1038\/mtm.2016.49_bib40","doi-asserted-by":"crossref","first-page":"483","DOI":"10.1089\/hum.2010.167","article-title":"Correction of hyperbilirubinemia in gunn rats using clinically relevant low doses of helper-dependent adenoviral vectors","volume":"22","author":"Dimmock","year":"2011","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mtm.2016.49_bib41","doi-asserted-by":"crossref","first-page":"999","DOI":"10.1053\/j.gastro.2010.05.008","article-title":"Lentiviral vectors that express UGT1A1 in liver and contain miR-142 target sequences normalize hyperbilirubinemia in Gunn rats","volume":"139","author":"Schmitt","year":"2010","journal-title":"Gastroenterol"},{"key":"10.1038\/mtm.2016.49_bib42","doi-asserted-by":"crossref","first-page":"957","DOI":"10.1182\/blood-2011-09-377630","article-title":"AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage","volume":"119","author":"Binny","year":"2012","journal-title":"Blood"},{"key":"10.1038\/mtm.2016.49_bib43","doi-asserted-by":"crossref","first-page":"280","DOI":"10.1038\/sj.mt.6300355","article-title":"Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose","volume":"16","author":"Wu","year":"2008","journal-title":"Mol Ther"},{"key":"10.1038\/mtm.2016.49_bib44","doi-asserted-by":"crossref","first-page":"974","DOI":"10.1111\/mmi.13079","article-title":"Nonoptimal codon usage influences protein structure in intrinsically disordered regions","volume":"97","author":"Zhou","year":"2015","journal-title":"Mol Microbiol"},{"key":"10.1038\/mtm.2016.49_bib45","first-page":"49","article-title":"Viral expression cassette elements to enhance transgene target specificity and expression in gene therapy","volume":"19","author":"Powell","year":"2015","journal-title":"Discov Med"},{"key":"10.1038\/mtm.2016.49_bib46","doi-asserted-by":"crossref","first-page":"1319","DOI":"10.1084\/jem.184.4.1319","article-title":"Ribosomal scanning past the primary initiation codon as a mechanism for expression of CTL epitopes encoded in alternative reading frames","volume":"184","author":"Bullock","year":"1996","journal-title":"J Exp Med"},{"key":"10.1038\/mtm.2016.49_bib47","doi-asserted-by":"crossref","first-page":"419","DOI":"10.1038\/nm1549","article-title":"CD8(+) T-cell responses to adeno-associated virus capsid in humans","volume":"13","author":"Mingozzi","year":"2007","journal-title":"Nat Med"},{"key":"10.1038\/mtm.2016.49_bib48","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":"10.1038\/mtm.2016.49_bib49","doi-asserted-by":"crossref","first-page":"382","DOI":"10.1038\/nature12875","article-title":"Selection and evaluation of clinically relevant AAV variants in a xenograft liver model","volume":"506","author":"Lisowski","year":"2014","journal-title":"Nature"},{"key":"10.1038\/mtm.2016.49_bib50","doi-asserted-by":"crossref","first-page":"1042","DOI":"10.1038\/mt.2016.61","article-title":"Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid","volume":"24","author":"Vercauteren","year":"2016","journal-title":"Mol Ther"},{"key":"10.1038\/mtm.2016.49_bib51","doi-asserted-by":"crossref","first-page":"1056","DOI":"10.1016\/j.celrep.2015.07.019","article-title":"In silico reconstruction of the viral evolutionary lineage yields a potent gene therapy vector","volume":"12","author":"Zinn","year":"2015","journal-title":"Cell Rep"},{"key":"10.1038\/mtm.2016.49_bib52","doi-asserted-by":"crossref","first-page":"7689","DOI":"10.1128\/JVI.77.13.7689-7695.2003","article-title":"Episomal persistence of recombinant adenoviral vector genomes during the cell cycle in vivo","volume":"77","author":"Ehrhardt","year":"2003","journal-title":"J Virol"},{"key":"10.1038\/mtm.2016.49_bib53","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":"10.1038\/mtm.2016.49_bib54","first-page":"55","article-title":"Body length and organ weights of infants and children: a study of the body length and normal weights of the more important vital organs of the body between birth and twelve years of age","volume":"9","author":"Coppoletta","year":"1933","journal-title":"Am J Pathol"},{"key":"10.1038\/mtm.2016.49_bib55","doi-asserted-by":"crossref","first-page":"1414","DOI":"10.1182\/blood-2006-03-010181","article-title":"Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates","volume":"109","author":"Nathwani","year":"2007","journal-title":"Blood"},{"key":"10.1038\/mtm.2016.49_bib56","doi-asserted-by":"crossref","first-page":"S34","DOI":"10.1016\/S1525-0016(16)32886-6","article-title":"Antigen-specific modulation of capsid immunogenicity with tolerogenic nanoparticles results in successful AAV vector readministration","volume":"24","author":"Meliani","year":"2016","journal-title":"Mol Ther"},{"key":"10.1038\/mtm.2016.49_bib57","doi-asserted-by":"crossref","first-page":"217","DOI":"10.1038\/nature10177","article-title":"In vivo genome editing restores haemostasis in a mouse model of haemophilia","volume":"475","author":"Li","year":"2011","journal-title":"Nature"},{"key":"10.1038\/mtm.2016.49_bib58","doi-asserted-by":"crossref","first-page":"3283","DOI":"10.1182\/blood-2013-04-497354","article-title":"Robust ZFN-mediated genome editing in adult hemophilic mice","volume":"122","author":"Anguela","year":"2013","journal-title":"Blood"},{"key":"10.1038\/mtm.2016.49_bib59","doi-asserted-by":"crossref","first-page":"360","DOI":"10.1038\/nature13864","article-title":"Promoterless gene targeting without nucleases ameliorates haemophilia B in mice","volume":"517","author":"Barzel","year":"2015","journal-title":"Nature"},{"key":"10.1038\/mtm.2016.49_bib60","first-page":"435","article-title":"Construction of adenovirus expression vectors by site-directed in vivo recombination","volume":"1","author":"Thummel","year":"1982","journal-title":"J Mol Appl Genet"},{"key":"10.1038\/mtm.2016.49_bib61","doi-asserted-by":"crossref","first-page":"503","DOI":"10.1038\/gt.2009.157","article-title":"High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency","volume":"17","author":"Ayuso","year":"2010","journal-title":"Gene Ther"},{"key":"10.1038\/mtm.2016.49_bib62","doi-asserted-by":"crossref","first-page":"938","DOI":"10.1038\/sj.gt.3300680","article-title":"Adeno-associated virus vectors can be efficiently produced without helper virus","volume":"5","author":"Matsushita","year":"1998","journal-title":"Gene Ther"},{"key":"10.1038\/mtm.2016.49_bib63","doi-asserted-by":"crossref","first-page":"2042","DOI":"10.1261\/rna.637907","article-title":"Substitution of the use of radioactivity by fluorescence for biochemical studies of RNA","volume":"13","author":"Ying","year":"2007","journal-title":"RNA"},{"key":"10.1038\/mtm.2016.49_bib64","first-page":"369","article-title":"Animal model of human disease. Crigler-Najjar Syndrome. Animal model: hereditary nonhemolytic unconjugated hyperbilirubinemia in Gunn rats","volume":"69","author":"Cornelius","year":"1972","journal-title":"Am J Pathol"},{"key":"10.1038\/mtm.2016.49_bib65","doi-asserted-by":"crossref","first-page":"e82597","DOI":"10.1371\/journal.pone.0082597","article-title":"Adeno-associated viral vector serotype 5 poorly transduces liver in rat models","volume":"8","author":"Montenegro-Miranda","year":"2013","journal-title":"PLoS One"}],"container-title":["Molecular Therapy - Methods &amp; Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050117300219?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050117300219?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2022,7,3]],"date-time":"2022-07-03T09:48:01Z","timestamp":1656841681000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050117300219"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2016]]},"references-count":65,"alternative-id":["S2329050117300219"],"URL":"https:\/\/doi.org\/10.1038\/mtm.2016.49","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2016]]},"article-number":"16049"}}