{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,6]],"date-time":"2026-03-06T15:49:15Z","timestamp":1772812155722,"version":"3.50.1"},"reference-count":92,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2019,12,1]],"date-time":"2019-12-01T00:00:00Z","timestamp":1575158400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2019,12,1]],"date-time":"2019-12-01T00:00:00Z","timestamp":1575158400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"}],"funder":[{"DOI":"10.13039\/100007249","name":"University of Minnesota","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007249","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Drug Discovery Today: Technologies"],"published-print":{"date-parts":[[2019,12]]},"DOI":"10.1016\/j.ddtec.2020.11.001","type":"journal-article","created":{"date-parts":[[2020,11,21]],"date-time":"2020-11-21T23:31:17Z","timestamp":1606001477000},"page":"9-19","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":6,"special_numbering":"C","title":["Liver targeted gene therapy: Insights into emerging therapies"],"prefix":"10.1016","volume":"34","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4469-0691","authenticated-orcid":false,"given":"Carlos G.","family":"Moscoso","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Clifford J.","family":"Steer","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"issue":"4","key":"10.1016\/j.ddtec.2020.11.001_bib0005","doi-asserted-by":"crossref","first-page":"501","DOI":"10.1016\/S0092-8674(00)80436-5","article-title":"Molecular reconstruction of Sleeping Beauty, a Tc1-like transposon from fish, and its transposition in human cells","volume":"91","author":"Ivics","year":"1997","journal-title":"Cell"},{"issue":"R1","key":"10.1016\/j.ddtec.2020.11.001_bib0010","doi-asserted-by":"crossref","first-page":"R14","DOI":"10.1093\/hmg\/ddr140","article-title":"The Sleeping Beauty transposon system: a non-viral vector for gene therapy","volume":"20","author":"Aronovich","year":"2011","journal-title":"Hum Mol Genet"},{"issue":"7","key":"10.1016\/j.ddtec.2020.11.001_bib0015","doi-asserted-by":"crossref","first-page":"2691","DOI":"10.1182\/blood-2004-09-3496","article-title":"Phenotypic correction and long-term expression of factor VIII in hemophilic mice by immunotolerization and nonviral gene transfer using the Sleeping Beauty transposon system","volume":"105","author":"Ohlfest","year":"2005","journal-title":"Blood"},{"issue":"7","key":"10.1016\/j.ddtec.2020.11.001_bib0020","first-page":"2086","article-title":"Nanocapsule-delivered Sleeping Beauty mediates therapeutic Factor VIII expression in liver sinusoidal endothelial cells of hemophilia A mice","volume":"119","author":"Kren","year":"2009","journal-title":"J Clin Invest"},{"issue":"1","key":"10.1016\/j.ddtec.2020.11.001_bib0025","doi-asserted-by":"crossref","first-page":"35","DOI":"10.1038\/75568","article-title":"Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system","volume":"25","author":"Yant","year":"2000","journal-title":"Nat Genet"},{"issue":"4","key":"10.1016\/j.ddtec.2020.11.001_bib0030","doi-asserted-by":"crossref","first-page":"654","DOI":"10.1016\/S1525-0016(03)00216-8","article-title":"Helper-independent Sleeping Beauty transposon-transposase vectors for efficient nonviral gene delivery and persistent gene expression in vivo","volume":"8","author":"Mikkelsen","year":"2003","journal-title":"Mol Ther"},{"issue":"5","key":"10.1016\/j.ddtec.2020.11.001_bib0035","doi-asserted-by":"crossref","first-page":"403","DOI":"10.1002\/jgm.1028","article-title":"Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses","volume":"9","author":"Aronovich","year":"2007","journal-title":"J Gene Med"},{"issue":"7","key":"10.1016\/j.ddtec.2020.11.001_bib0040","doi-asserted-by":"crossref","first-page":"1280","DOI":"10.1038\/sj.mt.6300160","article-title":"Messenger RNA as a source of transposase for Sleeping Beauty transposon-mediated correction of hereditary tyrosinemia type I","volume":"15","author":"Wilber","year":"2007","journal-title":"Mol Ther"},{"issue":"3","key":"10.1016\/j.ddtec.2020.11.001_bib0045","doi-asserted-by":"crossref","first-page":"620","DOI":"10.1038\/mt.2015.221","article-title":"Sleeping Beauty transposon vectors in liver-directed gene delivery of LDLR and VLDLR for gene therapy of familial hypercholesterolemia","volume":"24","author":"Turunen","year":"2016","journal-title":"Mol Ther"},{"issue":"3","key":"10.1016\/j.ddtec.2020.11.001_bib0050","doi-asserted-by":"crossref","first-page":"592","DOI":"10.1111\/jth.13938","article-title":"High and long-term von Willebrand factor expression after Sleeping Beauty transposon-mediated gene therapy in a mouse model of severe von Willebrand disease","volume":"16","author":"Portier","year":"2018","journal-title":"J Thromb Haemost"},{"issue":"7","key":"10.1016\/j.ddtec.2020.11.001_bib0055","doi-asserted-by":"crossref","first-page":"541","DOI":"10.1089\/hum.2017.003","article-title":"Transgene expression in dogs after liver-directed hydrodynamic delivery of Sleeping Beauty transposons using balloon catheters","volume":"28","author":"Hyland","year":"2017","journal-title":"Hum Gene Ther"},{"issue":"4","key":"10.1016\/j.ddtec.2020.11.001_bib0060","doi-asserted-by":"crossref","DOI":"10.1016\/j.gie.2018.06.022","article-title":"Successful liver-directed gene delivery by ERCP-guided hydrodynamic injection (with videos)","volume":"88","author":"Kumbhari","year":"2018","journal-title":"Gastrointest Endosc"},{"key":"10.1016\/j.ddtec.2020.11.001_bib0065","series-title":"Retroviruses","author":"Coffin","year":"1997"},{"issue":"9","key":"10.1016\/j.ddtec.2020.11.001_bib0070","doi-asserted-by":"crossref","first-page":"1805","DOI":"10.1111\/j.1538-7836.2007.02629.x","article-title":"High expression reduces an antibody response after neonatal gene therapy with B domain-deleted human factor VIII in mice","volume":"5","author":"Xu","year":"2007","journal-title":"J Thromb Haemost"},{"issue":"9","key":"10.1016\/j.ddtec.2020.11.001_bib0075","doi-asserted-by":"crossref","first-page":"794","DOI":"10.1002\/bies.201300049","article-title":"Are human endogenous retroviruses pathogenic? An approach to testing the hypothesis","volume":"35","author":"Young","year":"2013","journal-title":"Bioessays"},{"issue":"5","key":"10.1016\/j.ddtec.2020.11.001_bib0080","doi-asserted-by":"crossref","first-page":"1696","DOI":"10.1002\/hep.24230","article-title":"Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk","volume":"53","author":"M\u00e1trai","year":"2011","journal-title":"Hepatology"},{"key":"10.1016\/j.ddtec.2020.11.001_bib0085","doi-asserted-by":"crossref","first-page":"196","DOI":"10.1016\/j.virol.2014.12.001","article-title":"Suppression of hepatitis B virus DNA accumulation in chronically infected cells using a bacterial CRISPR\/Cas RNA-guided DNA endonuclease","volume":"476","author":"Kennedy","year":"2015","journal-title":"Virology"},{"issue":"8","key":"10.1016\/j.ddtec.2020.11.001_bib0090","doi-asserted-by":"crossref","first-page":"2252","DOI":"10.1099\/vir.0.000159","article-title":"Inhibition of hepatitis B virus by the CRISPR\/Cas9 system via targeting the conserved regions of the viral genome","volume":"96","author":"Liu","year":"2015","journal-title":"J Gen Virol"},{"issue":"4","key":"10.1016\/j.ddtec.2020.11.001_bib0095","doi-asserted-by":"crossref","first-page":"2004","DOI":"10.1128\/jvi.69.4.2004-2015.1995","article-title":"Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses","volume":"69","author":"Yang","year":"1995","journal-title":"J Virol"},{"issue":"22","key":"10.1016\/j.ddtec.2020.11.001_bib0100","doi-asserted-by":"crossref","first-page":"12816","DOI":"10.1073\/pnas.96.22.12816","article-title":"Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons","volume":"96","author":"Morral","year":"1999","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"3","key":"10.1016\/j.ddtec.2020.11.001_bib0105","doi-asserted-by":"crossref","first-page":"181","DOI":"10.1089\/hgtb.2013.236","article-title":"Correction of hyperbilirubinemia in Gunn rats by surgical delivery of low doses of helper-dependent adenoviral vectors","volume":"25","author":"Schmitt","year":"2014","journal-title":"Hum Gene Ther Methods"},{"issue":"12","key":"10.1016\/j.ddtec.2020.11.001_bib0110","doi-asserted-by":"crossref","first-page":"1058","DOI":"10.1038\/gt.2014.78","article-title":"Adenoviral transfer of human aquaporin-1 gene to rat liver improves bile flow in estrogen-induced cholestasis","volume":"21","author":"Marrone","year":"2014","journal-title":"Gene Ther"},{"key":"10.1016\/j.ddtec.2020.11.001_bib0115","doi-asserted-by":"crossref","first-page":"307","DOI":"10.1016\/j.ebiom.2018.09.051","article-title":"Targeting hepatic miR-221\/222 for therapeutic intervention of nonalcoholic steatohepatitis in mice","volume":"37","author":"Jiang","year":"2018","journal-title":"EBioMedicine"},{"issue":"4","key":"10.1016\/j.ddtec.2020.11.001_bib0120","doi-asserted-by":"crossref","first-page":"583","DOI":"10.1128\/CMR.00008-08","article-title":"Gene therapy using adeno-associated virus vectors","volume":"21","author":"Daya","year":"2008","journal-title":"Clin Microbiol Rev"},{"issue":"12","key":"10.1016\/j.ddtec.2020.11.001_bib0125","doi-asserted-by":"crossref","first-page":"1023","DOI":"10.1089\/hum.2014.099","article-title":"Selective in vivo targeting of human liver tumors by optimized AAV3 vectors in a murine xenograft model","volume":"25","author":"Ling","year":"2014","journal-title":"Hum Gene Ther"},{"issue":"15","key":"10.1016\/j.ddtec.2020.11.001_bib0130","doi-asserted-by":"crossref","first-page":"6969","DOI":"10.1128\/JVI.75.15.6969-6976.2001","article-title":"Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo","volume":"75","author":"Nakai","year":"2001","journal-title":"J Virol"},{"issue":"2","key":"10.1016\/j.ddtec.2020.11.001_bib0135","doi-asserted-by":"crossref","first-page":"419","DOI":"10.1016\/j.jhep.2015.09.014","article-title":"Long-term metabolic correction of Wilson\u2019s disease in a murine model by gene therapy","volume":"64","author":"Murillo","year":"2016","journal-title":"J Hepatol"},{"issue":"1","key":"10.1016\/j.ddtec.2020.11.001_bib0140","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1038\/mt.2009.250","article-title":"AAV8-mediated gene therapy prevents induced biochemical attacks of acute intermittent porphyria and improves neuromotor function","volume":"18","author":"Yasuda","year":"2010","journal-title":"Mol Ther"},{"issue":"2","key":"10.1016\/j.ddtec.2020.11.001_bib0145","doi-asserted-by":"crossref","first-page":"243","DOI":"10.1038\/mt.2010.210","article-title":"Sustained enzymatic correction by rAAV-mediated liver gene therapy protects against induced motor neuropathy in acute porphyria mice","volume":"19","author":"Unzu","year":"2011","journal-title":"Mol Ther"},{"issue":"3","key":"10.1016\/j.ddtec.2020.11.001_bib0150","doi-asserted-by":"crossref","first-page":"617","DOI":"10.1016\/j.ymgme.2012.09.019","article-title":"Pre-clinical efficacy and dosing of an AAV8 vector expressing human methylmalonyl-CoA mutase in a murine model of methylmalonic acidemia (MMA)","volume":"107","author":"Chandler","year":"2012","journal-title":"Mol Genet Metab"},{"issue":"3","key":"10.1016\/j.ddtec.2020.11.001_bib0155","doi-asserted-by":"crossref","first-page":"153","DOI":"10.1089\/hum.2014.126","article-title":"Long-term sex-biased correction of circulating propionic acidemia disease markers by adeno-associated virus vectors","volume":"26","author":"Guenzel","year":"2015","journal-title":"Hum Gene Ther"},{"issue":"2","key":"10.1016\/j.ddtec.2020.11.001_bib0160","doi-asserted-by":"crossref","first-page":"114","DOI":"10.1002\/jgm.1543","article-title":"Complete restoration of phenylalanine oxidation in phenylketonuria mouse by a self-complementary adeno-associated virus vector","volume":"13","author":"Yagi","year":"2011","journal-title":"J Gene Med"},{"issue":"1","key":"10.1016\/j.ddtec.2020.11.001_bib0165","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 Hepatol"},{"issue":"6","key":"10.1016\/j.ddtec.2020.11.001_bib0170","doi-asserted-by":"crossref","first-page":"1076","DOI":"10.1038\/mt.2010.64","article-title":"Complete normalization of hepatic G6PD deficiency, in murine glycogen storage disease type Ia using gene therapy","volume":"18","author":"Yiu","year":"2010","journal-title":"Mol Ther"},{"issue":"7","key":"10.1016\/j.ddtec.2020.11.001_bib0175","doi-asserted-by":"crossref","first-page":"903","DOI":"10.1089\/hum.2009.157","article-title":"Adeno-associated virus-mediated correction of a canine model of glycogen storage disease type Ia","volume":"21","author":"Weinstein","year":"2010","journal-title":"Hum Gene Ther"},{"issue":"19","key":"10.1016\/j.ddtec.2020.11.001_bib0180","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"},{"issue":"15","key":"10.1016\/j.ddtec.2020.11.001_bib0185","doi-asserted-by":"crossref","first-page":"1777","DOI":"10.1182\/blood-2014-12-615492","article-title":"In vivo genome editing of the albumin locus as a platform for protein replacement therapy","volume":"126","author":"Sharma","year":"2015","journal-title":"Blood"},{"issue":"25","key":"10.1016\/j.ddtec.2020.11.001_bib0190","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"},{"issue":"6","key":"10.1016\/j.ddtec.2020.11.001_bib0195","doi-asserted-by":"crossref","first-page":"399","DOI":"10.1002\/rmv.1762","article-title":"Adeno-associated virus (AAV) vectors in gene therapy: immune challenges and strategies to circumvent them","volume":"23","author":"Hareendran","year":"2013","journal-title":"Rev Med Virol"},{"issue":"11","key":"10.1016\/j.ddtec.2020.11.001_bib0200","doi-asserted-by":"crossref","first-page":"e1002994","DOI":"10.1371\/journal.ppat.1002994","article-title":"A wolf in sheep\u2019s clothing: SV40 co-opts host genome maintenance proteins to replicate viral DNA","volume":"8","author":"Sowd","year":"2012","journal-title":"PLoS Pathog"},{"issue":"2","key":"10.1016\/j.ddtec.2020.11.001_bib0205","doi-asserted-by":"crossref","first-page":"227","DOI":"10.1006\/mthe.2002.0657","article-title":"Durability of transgene expression and vector integration: recombinant SV40-derived gene therapy vectors","volume":"6","author":"Strayer","year":"2002","journal-title":"Mol Ther"},{"issue":"8","key":"10.1016\/j.ddtec.2020.11.001_bib0210","doi-asserted-by":"crossref","first-page":"1022","DOI":"10.1038\/nbt1231","article-title":"Gene targeting in vivo by adeno-associated virus vectors","volume":"24","author":"Miller","year":"2006","journal-title":"Nat Biotechnol"},{"issue":"4","key":"10.1016\/j.ddtec.2020.11.001_bib0215","doi-asserted-by":"crossref","first-page":"1200","DOI":"10.1002\/hep.23481","article-title":"Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo","volume":"51","author":"Paulk","year":"2010","journal-title":"Hepatology"},{"issue":"2","key":"10.1016\/j.ddtec.2020.11.001_bib0220","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1038\/sj.gt.3300601","article-title":"Therapeutic gene targeting","volume":"5","author":"Y\u00e1\u00f1ez","year":"1998","journal-title":"Gene Ther"},{"issue":"10","key":"10.1016\/j.ddtec.2020.11.001_bib0225","doi-asserted-by":"crossref","first-page":"3355","DOI":"10.1073\/pnas.82.10.3355","article-title":"Relative rates of homologous and nonhomologous recombination in transfected DNA","volume":"82","author":"Roth","year":"1985","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"1-2","key":"10.1016\/j.ddtec.2020.11.001_bib0230","doi-asserted-by":"crossref","first-page":"95","DOI":"10.1016\/j.mrfmmm.2006.01.022","article-title":"Analysis of DNA double-strand break repair pathways in mice","volume":"614","author":"Brugmans","year":"2007","journal-title":"Mutat Res"},{"issue":"22","key":"10.1016\/j.ddtec.2020.11.001_bib0235","doi-asserted-by":"crossref","first-page":"1910","DOI":"10.1038\/sj.gt.3302096","article-title":"Correction\/mutation of acid alpha-D-glucosidase gene by modified single-stranded oligonucleotides: in vitro and in vivo studies","volume":"10","author":"Lu","year":"2003","journal-title":"Gene Ther"},{"issue":"10","key":"10.1016\/j.ddtec.2020.11.001_bib0240","doi-asserted-by":"crossref","first-page":"838","DOI":"10.1038\/sj.gt.3302228","article-title":"Targeted conversion of the transthyretin gene in vitro and in vivo","volume":"11","author":"Nakamura","year":"2004","journal-title":"Gene Ther"},{"key":"10.1016\/j.ddtec.2020.11.001_bib0245","series-title":"Correction of the orntithine transcarbamylase point mutation in neonatal spfash mice using single-stranded oligonucleotides","first-page":"S323","author":"Kren","year":"2008"},{"key":"10.1016\/j.ddtec.2020.11.001_bib0250","doi-asserted-by":"crossref","first-page":"89","DOI":"10.1007\/978-1-4939-0992-6_8","article-title":"Targeted genome modification via triple helix formation","volume":"1176","author":"Ricciardi","year":"2014","journal-title":"Methods Mol Biol"},{"issue":"4","key":"10.1016\/j.ddtec.2020.11.001_bib0255","doi-asserted-by":"crossref","first-page":"1176","DOI":"10.1093\/nar\/27.4.1176","article-title":"Chromosomal mutations induced by triplex-forming oligonucleotides in mammalian cells","volume":"27","author":"Vasquez","year":"1999","journal-title":"Nucleic Acids Res"},{"issue":"7","key":"10.1016\/j.ddtec.2020.11.001_bib0260","doi-asserted-by":"crossref","first-page":"520","DOI":"10.1038\/cgt.2011.21","article-title":"DNA triplex-mediated inhibition of MET leads to cell death and tumor regression in hepatoma","volume":"18","author":"Singhal","year":"2011","journal-title":"Cancer Gene Ther"},{"issue":"1","key":"10.1016\/j.ddtec.2020.11.001_bib0265","doi-asserted-by":"crossref","first-page":"289","DOI":"10.1128\/MCB.21.1.289-297.2001","article-title":"Stimulation of homologous recombination through targeted cleavage by chimeric nucleases","volume":"21","author":"Bibikova","year":"2001","journal-title":"Mol Cell Biol"},{"issue":"8","key":"10.1016\/j.ddtec.2020.11.001_bib0270","doi-asserted-by":"crossref","first-page":"967","DOI":"10.1038\/nbt1125","article-title":"Gene targeting using zinc finger nucleases","volume":"23","author":"Porteus","year":"2005","journal-title":"Nat Biotechnol"},{"issue":"7","key":"10.1016\/j.ddtec.2020.11.001_bib0275","doi-asserted-by":"crossref","first-page":"669","DOI":"10.2174\/138955708784567458","article-title":"Critical parameters for genome editing using zinc finger nucleases","volume":"8","author":"Camenisch","year":"2008","journal-title":"Mini Rev Med Chem"},{"issue":"18","key":"10.1016\/j.ddtec.2020.11.001_bib0280","doi-asserted-by":"crossref","first-page":"10570","DOI":"10.1073\/pnas.95.18.10570","article-title":"FokI dimerization is required for DNA cleavage","volume":"95","author":"Bitinaite","year":"1998","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"7355","key":"10.1016\/j.ddtec.2020.11.001_bib0285","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"},{"issue":"3","key":"10.1016\/j.ddtec.2020.11.001_bib0290","doi-asserted-by":"crossref","first-page":"e21","DOI":"10.1093\/nar\/gku1246","article-title":"Multi-kilobase homozygous targeted gene replacement in human induced pluripotent stem cells","volume":"43","author":"Byrne","year":"2015","journal-title":"Nucleic Acids Res"},{"issue":"8","key":"10.1016\/j.ddtec.2020.11.001_bib0295","doi-asserted-by":"crossref","first-page":"e104816","DOI":"10.1371\/journal.pone.0104816","article-title":"Generation of Ugt1-deficient murine liver cell lines using TALEN technology","volume":"9","author":"Porro","year":"2014","journal-title":"PLoS One"},{"issue":"16","key":"10.1016\/j.ddtec.2020.11.001_bib0300","doi-asserted-by":"crossref","first-page":"9233","DOI":"10.1128\/JVI.01428-14","article-title":"Hepatitis C virus entry is impaired by claudin-1 downregulation in diacylglycerol acyltransferase-1-deficient cells","volume":"88","author":"Sung","year":"2014","journal-title":"J Virol"},{"issue":"6","key":"10.1016\/j.ddtec.2020.11.001_bib0305","doi-asserted-by":"crossref","first-page":"1239","DOI":"10.1016\/j.cell.2017.11.032","article-title":"The biology of CRISPR-Cas: backward and forward","volume":"172","author":"Hille","year":"2018","journal-title":"Cell"},{"issue":"7340","key":"10.1016\/j.ddtec.2020.11.001_bib0310","doi-asserted-by":"crossref","first-page":"602","DOI":"10.1038\/nature09886","article-title":"CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III","volume":"471","author":"Deltcheva","year":"2011","journal-title":"Nature"},{"issue":"7385","key":"10.1016\/j.ddtec.2020.11.001_bib0315","doi-asserted-by":"crossref","first-page":"331","DOI":"10.1038\/nature10886","article-title":"RNA-guided genetic silencing systems in bacteria and archaea","volume":"482","author":"Wiedenheft","year":"2012","journal-title":"Nature"},{"key":"10.1016\/j.ddtec.2020.11.001_bib0320","doi-asserted-by":"crossref","first-page":"273","DOI":"10.1146\/annurev-genet-110410-132430","article-title":"CRISPR-Cas systems in bacteria and archaea: versatile small RNAs for adaptive defense and regulation","volume":"45","author":"Bhaya","year":"2011","journal-title":"Annu Rev Genet"},{"issue":"7439","key":"10.1016\/j.ddtec.2020.11.001_bib0325","doi-asserted-by":"crossref","first-page":"50","DOI":"10.1038\/495050a","article-title":"Biotechnology: rewriting a genome","volume":"495","author":"Charpentier","year":"2013","journal-title":"Nature"},{"issue":"6096","key":"10.1016\/j.ddtec.2020.11.001_bib0330","doi-asserted-by":"crossref","first-page":"816","DOI":"10.1126\/science.1225829","article-title":"A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity","volume":"337","author":"Jinek","year":"2012","journal-title":"Science"},{"issue":"7490","key":"10.1016\/j.ddtec.2020.11.001_bib0335","doi-asserted-by":"crossref","first-page":"62","DOI":"10.1038\/nature13011","article-title":"DNA interrogation by the CRISPR RNA-guided endonuclease Cas9","volume":"507","author":"Sternberg","year":"2014","journal-title":"Nature"},{"issue":"3","key":"10.1016\/j.ddtec.2020.11.001_bib0340","doi-asserted-by":"crossref","first-page":"230","DOI":"10.1038\/nbt.2507","article-title":"Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease","volume":"31","author":"Cho","year":"2013","journal-title":"Nat Biotechnol"},{"issue":"6121","key":"10.1016\/j.ddtec.2020.11.001_bib0345","doi-asserted-by":"crossref","first-page":"819","DOI":"10.1126\/science.1231143","article-title":"Multiplex genome engineering using CRISPR\/Cas systems","volume":"339","author":"Cong","year":"2013","journal-title":"Science"},{"issue":"6","key":"10.1016\/j.ddtec.2020.11.001_bib0350","doi-asserted-by":"crossref","first-page":"551","DOI":"10.1038\/nbt.2884","article-title":"Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype","volume":"32","author":"Yin","year":"2014","journal-title":"Nat Biotechnol"},{"issue":"7522","key":"10.1016\/j.ddtec.2020.11.001_bib0355","doi-asserted-by":"crossref","first-page":"380","DOI":"10.1038\/nature13589","article-title":"CRISPR-mediated direct mutation of cancer genes in the mouse liver","volume":"514","author":"Xue","year":"2014","journal-title":"Nature"},{"issue":"2","key":"10.1016\/j.ddtec.2020.11.001_bib0360","doi-asserted-by":"crossref","first-page":"440","DOI":"10.1016\/j.cell.2014.09.014","article-title":"CRISPR-Cas9 knockin mice for genome editing and cancer modeling","volume":"159","author":"Platt","year":"2014","journal-title":"Cell"},{"key":"10.1016\/j.ddtec.2020.11.001_bib0365","doi-asserted-by":"crossref","first-page":"44624","DOI":"10.1038\/srep44624","article-title":"Somatic genome editing with CRISPR\/Cas9 generates and corrects a metabolic disease","volume":"7","author":"Jarrett","year":"2017","journal-title":"Sci Rep"},{"issue":"1","key":"10.1016\/j.ddtec.2020.11.001_bib0370","doi-asserted-by":"crossref","first-page":"2585","DOI":"10.1038\/s41598-017-02927-2","article-title":"Proof-of-concept gene editing for the murine model of inducible arginase-1 deficiency","volume":"7","author":"Sin","year":"2017","journal-title":"Sci Rep"},{"issue":"2","key":"10.1016\/j.ddtec.2020.11.001_bib0375","doi-asserted-by":"crossref","first-page":"139","DOI":"10.1038\/s41434-018-0003-1","article-title":"Targeted in vivo knock-in of human alpha-1-antitrypsin cDNA using adenoviral delivery of CRISPR\/Cas9","volume":"25","author":"Stephens","year":"2018","journal-title":"Gene Ther"},{"key":"10.1016\/j.ddtec.2020.11.001_bib0380","doi-asserted-by":"crossref","first-page":"101467","DOI":"10.1016\/j.scr.2019.101467","article-title":"Generation of induced pluripotent stem cells-derived hepatocyte-like cells for ex vivo gene therapy of primary hyperoxaluria type 1","volume":"38","author":"Est\u00e8ve","year":"2019","journal-title":"Stem Cell Res"},{"issue":"4","key":"10.1016\/j.ddtec.2020.11.001_bib0385","doi-asserted-by":"crossref","first-page":"677","DOI":"10.1016\/j.bbrc.2019.07.109","article-title":"Targeted gene therapy in human-induced pluripotent stem cells from a patient with primary hyperoxaluria type 1 using CRISPR\/Cas9 technology","volume":"517","author":"Est\u00e8ve","year":"2019","journal-title":"Biochem Biophys Res Commun"},{"issue":"5","key":"10.1016\/j.ddtec.2020.11.001_bib0390","doi-asserted-by":"crossref","first-page":"7199","DOI":"10.3892\/mmr.2017.7518","article-title":"Efficient inhibition of duck hepatitis B virus DNA by the CRISPR\/Cas9 system","volume":"16","author":"Zheng","year":"2017","journal-title":"Mol Med Rep"},{"issue":"9","key":"10.1016\/j.ddtec.2020.11.001_bib0395","doi-asserted-by":"crossref","first-page":"1779","DOI":"10.1007\/s00018-019-03021-8","article-title":"Orthologous CRISPR\/Cas9 systems for specific and efficient degradation of covalently closed circular DNA of hepatitis B virus","volume":"76","author":"Kostyushev","year":"2019","journal-title":"Cell Mol Life Sci"},{"key":"10.1016\/j.ddtec.2020.11.001_bib0400","doi-asserted-by":"crossref","first-page":"58","DOI":"10.1016\/j.antiviral.2018.02.011","article-title":"Inhibition of hepatitis B virus replication via HBV DNA cleavage by Cas9 from Staphylococcus aureus","volume":"152","author":"Liu","year":"2018","journal-title":"Antiviral Res"},{"issue":"6","key":"10.1016\/j.ddtec.2020.11.001_bib0405","doi-asserted-by":"crossref","first-page":"355","DOI":"10.1089\/crispr.2018.29039.spr","article-title":"#CRISPRbabies: notes on a scandal","volume":"1","author":"Ryder","year":"2018","journal-title":"CRISPR J"},{"issue":"7681","key":"10.1016\/j.ddtec.2020.11.001_bib0410","doi-asserted-by":"crossref","first-page":"464","DOI":"10.1038\/nature24644","article-title":"Programmable base editing of A\u2022T to G\u2022C in genomic DNA without DNA cleavage","volume":"551","author":"Gaudelli","year":"2017","journal-title":"Nature"},{"issue":"7603","key":"10.1016\/j.ddtec.2020.11.001_bib0415","doi-asserted-by":"crossref","first-page":"420","DOI":"10.1038\/nature17946","article-title":"Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage","volume":"533","author":"Komor","year":"2016","journal-title":"Nature"},{"issue":"7785","key":"10.1016\/j.ddtec.2020.11.001_bib0420","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1038\/s41586-019-1711-4","article-title":"Search-and-replace genome editing without double-strand breaks or donor DNA","volume":"576","author":"Anzalone","year":"2019","journal-title":"Nature"},{"issue":"24","key":"10.1016\/j.ddtec.2020.11.001_bib0425","doi-asserted-by":"crossref","first-page":"e00850","DOI":"10.1128\/JVI.00850-17","article-title":"Atomic structures of minor proteins VI and VII in human adenovirus","volume":"91","author":"Dai","year":"2017","journal-title":"J Virol"},{"issue":"2","key":"10.1016\/j.ddtec.2020.11.001_bib0430","doi-asserted-by":"crossref","first-page":"308","DOI":"10.1016\/j.jsb.2014.03.020","article-title":"The structure of AAVrh32.33, a novel gene delivery vector","volume":"186","author":"Mikals","year":"2014","journal-title":"J Struct Biol"},{"issue":"2","key":"10.1016\/j.ddtec.2020.11.001_bib0435","doi-asserted-by":"crossref","first-page":"165","DOI":"10.1016\/S0969-2126(96)00020-2","article-title":"The structure of simian virus 40 refined at 3.1 \u00c5 resolution","volume":"4","author":"Stehle","year":"1996","journal-title":"Structure"},{"key":"10.1016\/j.ddtec.2020.11.001_bib0440","series-title":"Mol*: towards a common library and tools for web molecular graphics. Proceedings of the Workshop on Molecular Graphics and Visual Analysis of Molecular Data","first-page":"29","author":"Sehnal","year":"2018"},{"issue":"10","key":"10.1016\/j.ddtec.2020.11.001_bib0445","doi-asserted-by":"crossref","first-page":"1171","DOI":"10.1016\/S0969-2126(96)00125-6","article-title":"Zif268 protein-DNA complex refined at 1.6 A: a model system for understanding zinc finger-DNA interactions","volume":"4","author":"Elrod-Erickson","year":"1996","journal-title":"Structure"},{"issue":"6069","key":"10.1016\/j.ddtec.2020.11.001_bib0450","doi-asserted-by":"crossref","first-page":"716","DOI":"10.1126\/science.1216211","article-title":"The crystal structure of TAL effector PthXo1 bound to its DNA target","volume":"335","author":"Mak","year":"2012","journal-title":"Science"},{"issue":"1","key":"10.1016\/j.ddtec.2020.11.001_bib0455","doi-asserted-by":"crossref","first-page":"48","DOI":"10.1016\/j.cell.2017.06.012","article-title":"Structure basis for directional R-loop formation and substrate handover mechanisms in type I CRISPR-Cas system","volume":"170","author":"Xiao","year":"2017","journal-title":"Cell"},{"issue":"1","key":"10.1016\/j.ddtec.2020.11.001_bib0460","doi-asserted-by":"crossref","first-page":"14","DOI":"10.1002\/pro.3235","article-title":"UCSF ChimeraX: meeting modern challenges in visualization and analysis","volume":"27","author":"Goddard","year":"2018","journal-title":"Protein Sci"}],"container-title":["Drug Discovery Today: Technologies"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1740674920300226?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1740674920300226?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,28]],"date-time":"2025-10-28T16:21:39Z","timestamp":1761668499000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1740674920300226"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,12]]},"references-count":92,"alternative-id":["S1740674920300226"],"URL":"https:\/\/doi.org\/10.1016\/j.ddtec.2020.11.001","relation":{},"ISSN":["1740-6749"],"issn-type":[{"value":"1740-6749","type":"print"}],"subject":[],"published":{"date-parts":[[2019,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Liver targeted gene therapy: Insights into emerging therapies","name":"articletitle","label":"Article Title"},{"value":"Drug Discovery Today: Technologies","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ddtec.2020.11.001","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2020 Elsevier Ltd. All rights reserved.","name":"copyright","label":"Copyright"}]}}