{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,4]],"date-time":"2026-11-04T09:14:04Z","timestamp":1793783644827,"version":"4.6.1"},"reference-count":64,"publisher":"Elsevier BV","issue":"11","license":[{"start":{"date-parts":[[2021,11,1]],"date-time":"2021-11-01T00:00:00Z","timestamp":1635724800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2021,11,1]],"date-time":"2021-11-01T00:00:00Z","timestamp":1635724800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2021,3,23]],"date-time":"2021-03-23T00:00:00Z","timestamp":1616457600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2021,11]]},"DOI":"10.1016\/j.ymthe.2021.03.018","type":"journal-article","created":{"date-parts":[[2021,3,26]],"date-time":"2021-03-26T23:57:17Z","timestamp":1616803037000},"page":"3230-3242","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":24,"title":["ZFN-mediated in\u00a0vivo gene editing in hepatocytes leads to supraphysiologic \u03b1-Gal A activity and effective substrate reduction in Fabry mice"],"prefix":"10.1016","volume":"29","author":[{"given":"Silvere","family":"Pagant","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marshall W.","family":"Huston","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Luciana","family":"Moreira","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lin","family":"Gan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susan","family":"St Martin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Scott","family":"Sproul","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael C.","family":"Holmes","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathleen","family":"Meyer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas","family":"Wechsler","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert J.","family":"Desnick","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3255-7324","authenticated-orcid":false,"given":"Makiko","family":"Yasuda","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymthe.2021.03.018_bib1","series-title":"The Metabolic and Molecular Bases of Inherited Disease","first-page":"3733","article-title":"\u03b1-galactosidase A deficiency: Fabry disease","author":"Desnick","year":"2001"},{"key":"10.1016\/j.ymthe.2021.03.018_bib2","doi-asserted-by":"crossref","first-page":"2812","DOI":"10.1073\/pnas.0712309105","article-title":"Elevated globotriaosylsphingosine is a hallmark of Fabry disease","volume":"105","author":"Aerts","year":"2008","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2021.03.018_bib3","doi-asserted-by":"crossref","first-page":"705","DOI":"10.1007\/s00428-008-0614-9","article-title":"Cellular and tissue localization of globotriaosylceramide in Fabry disease","volume":"452","author":"Elleder","year":"2008","journal-title":"Virchows Arch."},{"key":"10.1016\/j.ymthe.2021.03.018_bib4","doi-asserted-by":"crossref","first-page":"2561","DOI":"10.1161\/CIRCULATIONAHA.108.841494","article-title":"Cardiac microvascular pathology in Fabry disease: evaluation of endomyocardial biopsies before and after enzyme replacement therapy","volume":"119","author":"Thurberg","year":"2009","journal-title":"Circulation"},{"key":"10.1016\/j.ymthe.2021.03.018_bib5","doi-asserted-by":"crossref","first-page":"1933","DOI":"10.1046\/j.1523-1755.2002.00675.x","article-title":"Globotriaosylceramide accumulation in the Fabry kidney is cleared from multiple cell types after enzyme replacement therapy","volume":"62","author":"Thurberg","year":"2002","journal-title":"Kidney Int."},{"issue":"Suppl 2","key":"10.1016\/j.ymthe.2021.03.018_bib6","first-page":"S172","article-title":"Fabry disease: clinical spectrum and evidence-based enzyme replacement therapy","volume":"2","author":"Desnick","year":"2006","journal-title":"Nephrol. Ther."},{"key":"10.1016\/j.ymthe.2021.03.018_bib7","first-page":"317","article-title":"Fabry disease: clinical features and recent advances in enzyme replacement therapy","volume":"31","author":"Desnick","year":"2001","journal-title":"Adv. Nephrol. Necker Hosp."},{"key":"10.1016\/j.ymthe.2021.03.018_bib8","doi-asserted-by":"crossref","first-page":"801","DOI":"10.1046\/j.1523-1755.2003.00160.x","article-title":"Fabry disease: detection of undiagnosed hemodialysis patients and identification of a \u201crenal variant\u201d phenotype","volume":"64","author":"Nakao","year":"2003","journal-title":"Kidney Int."},{"key":"10.1016\/j.ymthe.2021.03.018_bib9","doi-asserted-by":"crossref","first-page":"395","DOI":"10.1056\/NEJM199102073240607","article-title":"An atypical variant of Fabry\u2019s disease with manifestations confined to the myocardium","volume":"324","author":"von Scheidt","year":"1991","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.ymthe.2021.03.018_bib10","doi-asserted-by":"crossref","first-page":"288","DOI":"10.1056\/NEJM199508033330504","article-title":"An atypical variant of Fabry\u2019s disease in men with left ventricular hypertrophy","volume":"333","author":"Nakao","year":"1995","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.ymthe.2021.03.018_bib11","doi-asserted-by":"crossref","first-page":"44","DOI":"10.1111\/cge.12613","article-title":"X-chromosome inactivation in female patients with Fabry disease","volume":"89","author":"Echevarria","year":"2016","journal-title":"Clin. Genet."},{"key":"10.1016\/j.ymthe.2021.03.018_bib12","doi-asserted-by":"crossref","first-page":"711","DOI":"10.1086\/318809","article-title":"A phase 1\/2 clinical trial of enzyme replacement in fabry disease: pharmacokinetic, substrate clearance, and safety studies","volume":"68","author":"Eng","year":"2001","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.ymthe.2021.03.018_bib13","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1056\/NEJM200107053450102","article-title":"Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry\u2019s disease","volume":"345","author":"Eng","year":"2001","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.ymthe.2021.03.018_bib14","doi-asserted-by":"crossref","first-page":"353","DOI":"10.1136\/jmedgenet-2014-102797","article-title":"Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease","volume":"52","author":"Germain","year":"2015","journal-title":"J.\u00a0Med. Genet."},{"key":"10.1016\/j.ymthe.2021.03.018_bib15","doi-asserted-by":"crossref","first-page":"338","DOI":"10.7326\/0003-4819-138-4-200302180-00014","article-title":"Fabry disease, an under-recognized multisystemic disorder: expert recommendations for diagnosis, management, and enzyme replacement therapy","volume":"138","author":"Desnick","year":"2003","journal-title":"Ann. Intern. Med."},{"key":"10.1016\/j.ymthe.2021.03.018_bib16","first-page":"100454","article-title":"The effect of enzyme replacement therapy on clinical outcomes in male patients with Fabry disease: A systematic literature review by a European panel of experts","volume":"19","author":"Germain","year":"2019","journal-title":"Mol. Genet. Metab. Rep."},{"key":"10.1016\/j.ymthe.2021.03.018_bib17","doi-asserted-by":"crossref","first-page":"416","DOI":"10.1016\/j.ymgme.2018.02.014","article-title":"Fabry disease revisited: Management and treatment recommendations for adult patients","volume":"123","author":"Ortiz","year":"2018","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2021.03.018_bib18","doi-asserted-by":"crossref","first-page":"256","DOI":"10.1681\/ASN.2014121226","article-title":"Serum-Mediated Inhibition of Enzyme Replacement Therapy in Fabry Disease","volume":"27","author":"Lenders","year":"2016","journal-title":"J.\u00a0Am. Soc. Nephrol."},{"key":"10.1016\/j.ymthe.2021.03.018_bib19","doi-asserted-by":"crossref","first-page":"1589","DOI":"10.1111\/j.1523-1755.2004.00924.x","article-title":"Enzyme therapy for Fabry disease: neutralizing antibodies toward agalsidase alpha and beta","volume":"66","author":"Linthorst","year":"2004","journal-title":"Kidney Int."},{"key":"10.1016\/j.ymthe.2021.03.018_bib20","doi-asserted-by":"crossref","first-page":"430","DOI":"10.1038\/gim.2016.122","article-title":"The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat","volume":"19","author":"Benjamin","year":"2017","journal-title":"Genet. Med."},{"key":"10.1016\/j.ymthe.2021.03.018_bib21","doi-asserted-by":"crossref","first-page":"e1003632","DOI":"10.1371\/journal.pgen.1003632","article-title":"Functional characterisation of alpha-galactosidase a mutations as a basis for a new classification system in fabry disease","volume":"9","author":"Lukas","year":"2013","journal-title":"PLoS Genet."},{"key":"10.1016\/j.ymthe.2021.03.018_bib22","doi-asserted-by":"crossref","first-page":"965","DOI":"10.1002\/humu.21530","article-title":"A pharmacogenetic approach to identify mutant forms of \u03b1-galactosidase A that respond to a pharmacological chaperone for Fabry disease","volume":"32","author":"Wu","year":"2011","journal-title":"Hum. Mutat."},{"key":"10.1016\/j.ymthe.2021.03.018_bib23","doi-asserted-by":"crossref","first-page":"4856","DOI":"10.1038\/s41467-018-07310-x","article-title":"Orthogonal Cas9-Cas9 chimeras provide a versatile platform for genome editing","volume":"9","author":"Bolukbasi","year":"2018","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2021.03.018_bib24","doi-asserted-by":"crossref","first-page":"52","DOI":"10.1016\/j.tcb.2015.07.009","article-title":"Repair Pathway Choices and Consequences at the Double-Strand Break","volume":"26","author":"Ceccaldi","year":"2016","journal-title":"Trends Cell Biol."},{"key":"10.1016\/j.ymthe.2021.03.018_bib25","doi-asserted-by":"crossref","first-page":"497","DOI":"10.1016\/j.molcel.2012.07.029","article-title":"Playing the end game: DNA double-strand break repair pathway choice","volume":"47","author":"Chapman","year":"2012","journal-title":"Mol. Cell"},{"key":"10.1016\/j.ymthe.2021.03.018_bib26","doi-asserted-by":"crossref","first-page":"764","DOI":"10.1126\/science.1079512","article-title":"Enhancing gene targeting with designed zinc finger nucleases","volume":"300","author":"Bibikova","year":"2003","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2021.03.018_bib27","doi-asserted-by":"crossref","first-page":"1156","DOI":"10.1073\/pnas.93.3.1156","article-title":"Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain","volume":"93","author":"Kim","year":"1996","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2021.03.018_bib28","doi-asserted-by":"crossref","first-page":"3361","DOI":"10.1093\/nar\/28.17.3361","article-title":"Requirements for double-strand cleavage by chimeric restriction enzymes with zinc finger DNA-recognition domains","volume":"28","author":"Smith","year":"2000","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.ymthe.2021.03.018_bib29","doi-asserted-by":"crossref","first-page":"14","DOI":"10.1086\/316953","article-title":"Fabry disease: preclinical studies demonstrate the effectiveness of alpha-galactosidase A replacement in enzyme-deficient mice","volume":"68","author":"Ioannou","year":"2001","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.ymthe.2021.03.018_bib30","doi-asserted-by":"crossref","first-page":"1127","DOI":"10.1016\/j.ymthe.2018.03.002","article-title":"Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In\u00a0Vivo Genome Editing","volume":"26","author":"Laoharawee","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2021.03.018_bib31","doi-asserted-by":"crossref","first-page":"178","DOI":"10.1016\/j.ymthe.2018.10.018","article-title":"ZFN-Mediated In\u00a0Vivo Genome Editing Corrects Murine Hurler Syndrome","volume":"27","author":"Ou","year":"2019","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2021.03.018_bib32","doi-asserted-by":"crossref","first-page":"1777","DOI":"10.1182\/blood-2014-12-615492","article-title":"In\u00a0vivo genome editing of the albumin locus as a platform for protein replacement therapy","volume":"126","author":"Sharma","year":"2015","journal-title":"Blood"},{"key":"10.1016\/j.ymthe.2021.03.018_bib33","doi-asserted-by":"crossref","first-page":"589","DOI":"10.1038\/nbt957","article-title":"Correction of multi-gene deficiency in\u00a0vivo using a single \u2018self-cleaving\u2019 2A peptide-based retroviral vector","volume":"22","author":"Szymczak","year":"2004","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2021.03.018_bib34","doi-asserted-by":"crossref","first-page":"2062","DOI":"10.1016\/S0021-9258(18)61618-7","article-title":"Synthesis and processing of alpha-galactosidase A in human fibroblasts. Evidence for different mutations in Fabry disease","volume":"262","author":"Lemansky","year":"1987","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.ymthe.2021.03.018_bib35","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":"10.1016\/j.ymthe.2021.03.018_bib36","doi-asserted-by":"crossref","first-page":"1390","DOI":"10.1128\/JB.01412-07","article-title":"Phage response to CRISPR-encoded resistance in Streptococcus thermophilus","volume":"190","author":"Deveau","year":"2008","journal-title":"J.\u00a0Bacteriol."},{"key":"10.1016\/j.ymthe.2021.03.018_bib37","doi-asserted-by":"crossref","first-page":"733","DOI":"10.1099\/mic.0.023960-0","article-title":"Short motif sequences determine the targets of the prokaryotic CRISPR defence system","volume":"155","author":"Mojica","year":"2009","journal-title":"Microbiology (Reading)"},{"key":"10.1016\/j.ymthe.2021.03.018_bib38","doi-asserted-by":"crossref","first-page":"183","DOI":"10.1358\/dof.2012.37.3.1779022","article-title":"Zinc Finger Nucleases: Tailor-made for Gene Therapy","volume":"37","author":"Chou","year":"2012","journal-title":"Drugs Future"},{"key":"10.1016\/j.ymthe.2021.03.018_bib39","doi-asserted-by":"crossref","first-page":"945","DOI":"10.1038\/s41587-019-0186-z","article-title":"Enhancing gene editing specificity by attenuating DNA cleavage kinetics","volume":"37","author":"Miller","year":"2019","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2021.03.018_bib40","doi-asserted-by":"crossref","first-page":"74","DOI":"10.1038\/nmeth.1539","article-title":"Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures","volume":"8","author":"Doyon","year":"2011","journal-title":"Nat. Methods"},{"key":"10.1016\/j.ymthe.2021.03.018_bib41","doi-asserted-by":"crossref","first-page":"778","DOI":"10.1038\/nbt1319","article-title":"An improved zinc-finger nuclease architecture for highly specific genome editing","volume":"25","author":"Miller","year":"2007","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2021.03.018_bib42","doi-asserted-by":"crossref","first-page":"1133","DOI":"10.1038\/s41467-019-08867-x","article-title":"Diversifying the structure of zinc finger nucleases for high-precision genome editing","volume":"10","author":"Paschon","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2021.03.018_bib43","doi-asserted-by":"crossref","first-page":"1234","DOI":"10.1080\/10717544.2018.1474964","article-title":"Delivering CRISPR: a review of the challenges and approaches","volume":"25","author":"Lino","year":"2018","journal-title":"Drug Deliv."},{"key":"10.1016\/j.ymthe.2021.03.018_bib44","doi-asserted-by":"crossref","first-page":"3781","DOI":"10.1016\/S0021-9258(18)53762-5","article-title":"Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter","volume":"268","author":"Flotte","year":"1993","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.ymthe.2021.03.018_bib45","doi-asserted-by":"crossref","first-page":"1267","DOI":"10.1038\/ng.3893","article-title":"Identification of liver-specific enhancer-promoter activity in the 3\u2032 untranslated region of the wild-type AAV2 genome","volume":"49","author":"Logan","year":"2017","journal-title":"Nat. Genet."},{"key":"10.1016\/j.ymthe.2021.03.018_bib46","doi-asserted-by":"crossref","first-page":"286","DOI":"10.1002\/jcp.21172","article-title":"Liver regeneration","volume":"213","author":"Michalopoulos","year":"2007","journal-title":"J.\u00a0Cell. Physiol."},{"key":"10.1016\/j.ymthe.2021.03.018_bib47","doi-asserted-by":"crossref","first-page":"620","DOI":"10.1016\/j.dnarep.2013.04.015","article-title":"DNA repair mechanisms in dividing and non-dividing cells","volume":"12","author":"Iyama","year":"2013","journal-title":"DNA Repair (Amst.)"},{"key":"10.1016\/j.ymthe.2021.03.018_bib48","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1038\/s41587-020-0741-7","article-title":"A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells","volume":"39","author":"Nguyen","year":"2021","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2021.03.018_bib49","doi-asserted-by":"crossref","first-page":"34","DOI":"10.1016\/j.ymthe.2006.03.008","article-title":"Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver","volume":"14","author":"Bell","year":"2006","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2021.03.018_bib50","doi-asserted-by":"crossref","first-page":"870","DOI":"10.1172\/JCI79213","article-title":"Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy","volume":"125","author":"Chandler","year":"2015","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.ymthe.2021.03.018_bib51","doi-asserted-by":"crossref","first-page":"2098","DOI":"10.1038\/mt.2012.197","article-title":"Patterns of scAAV vector insertion associated with oncogenic events in a mouse model for genotoxicity","volume":"20","author":"Rosas","year":"2012","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2021.03.018_bib52","doi-asserted-by":"crossref","first-page":"1100","DOI":"10.1038\/mt.2016.52","article-title":"Recombinant AAV Integration Is Not Associated With Hepatic Genotoxicity in Nonhuman Primates and Patients","volume":"24","author":"Gil-Farina","year":"2016","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2021.03.018_bib53","doi-asserted-by":"crossref","first-page":"91","DOI":"10.1016\/j.ymgme.2008.10.017","article-title":"Long-term inhibition of glycosphingolipid accumulation in Fabry model mice by a single systemic injection of AAV1 vector in the neonatal period","volume":"96","author":"Ogawa","year":"2009","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2021.03.018_bib54","doi-asserted-by":"crossref","first-page":"607","DOI":"10.1016\/j.omtm.2020.07.002","article-title":"AAV2\/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction","volume":"18","author":"Yasuda","year":"2020","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2021.03.018_bib55","doi-asserted-by":"crossref","first-page":"492","DOI":"10.1038\/sj.mt.6300066","article-title":"Correction of the Biochemical and Functional Deficits in Fabry Mice Following AAV8-mediated Hepatic Expression of alpha-galactosidase A","volume":"15","author":"Ziegler","year":"2007","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2021.03.018_bib56","doi-asserted-by":"crossref","first-page":"231","DOI":"10.1016\/j.ymthe.2003.11.015","article-title":"AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice","volume":"9","author":"Ziegler","year":"2004","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2021.03.018_bib57","first-page":"S68","article-title":"EMPOWERS: A phase 1\/2 clinical trial of SB-318 ZFN-mediated in\u00a0vivo human genome editing for treatment of MPS I (Hurler syndrome)","volume":"126","author":"Harmatz","year":"2019","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2021.03.018_bib58","first-page":"S104","article-title":"CHAMPIONS: A phase 1\/2 clinical trial with dose escalation of SB-913 ZFN-mediated in\u00a0vivo human genome editing for treatment of MPS II (Hunter syndrome)","volume":"126","author":"Muenzer","year":"2019","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2021.03.018_bib59","doi-asserted-by":"crossref","first-page":"5085","DOI":"10.1128\/JVI.72.6.5085-5092.1998","article-title":"Woodchuck hepatitis virus contains a tripartite posttranscriptional regulatory element","volume":"72","author":"Donello","year":"1998","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.ymthe.2021.03.018_bib60","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":"10.1016\/j.ymthe.2021.03.018_bib61","series-title":"Methods in Molecular Biology","doi-asserted-by":"crossref","first-page":"247","DOI":"10.1007\/978-1-61779-095-9_11","article-title":"Vector characterization methods for quality control testing of recombinant adeno-associated viruses","author":"Wright","year":"2011"},{"key":"10.1016\/j.ymthe.2021.03.018_bib62","first-page":"A208","article-title":"Generation of a mouse model with \u03b1-galactosidase A deficiency","volume":"59","author":"Wang","year":"1996","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.ymthe.2021.03.018_bib63","first-page":"157","article-title":"Fabry\u2019s disease: enzymatic diagnosis of hemizygotes and heterozygotes. Alpha-galactosidase activities in plasma, serum, urine, and leukocytes","volume":"81","author":"Desnick","year":"1973","journal-title":"J.\u00a0Lab. Clin. Med."},{"key":"10.1016\/j.ymthe.2021.03.018_bib64","doi-asserted-by":"crossref","first-page":"247","DOI":"10.1016\/0009-8981(81)90384-3","article-title":"Differential assay for lysosomal alpha-galactosidases in human tissues and its application to Fabry\u2019s disease","volume":"112","author":"Mayes","year":"1981","journal-title":"Clin. Chim. Acta"}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001621001477?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001621001477?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2024,5,1]],"date-time":"2024-05-01T22:52:58Z","timestamp":1714603978000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001621001477"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,11]]},"references-count":64,"journal-issue":{"issue":"11","published-print":{"date-parts":[[2021,11]]}},"alternative-id":["S1525001621001477"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2021.03.018","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2021,11]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"ZFN-mediated in\u00a0vivo gene editing in hepatocytes leads to supraphysiologic \u03b1-Gal A activity and effective substrate reduction in Fabry mice","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2021.03.018","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2021 The Author(s).","name":"copyright","label":"Copyright"}]}}