{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,29]],"date-time":"2026-09-29T14:16:52Z","timestamp":1790691412537,"version":"4.1.0"},"reference-count":52,"publisher":"Elsevier BV","issue":"10","license":[{"start":{"date-parts":[[2020,10,1]],"date-time":"2020-10-01T00:00:00Z","timestamp":1601510400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2020,10,1]],"date-time":"2020-10-01T00:00:00Z","timestamp":1601510400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2021,10,7]],"date-time":"2021-10-07T00:00:00Z","timestamp":1633564800000},"content-version":"vor","delay-in-days":371,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2020,10]]},"DOI":"10.1016\/j.ymthe.2020.06.021","type":"journal-article","created":{"date-parts":[[2020,6,18]],"date-time":"2020-06-18T21:55:23Z","timestamp":1592517323000},"page":"2150-2160","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":38,"title":["Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice"],"prefix":"10.1016","volume":"28","author":[{"given":"Hannah G.","family":"Lahey","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chelsea J.","family":"Webber","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diane","family":"Golebiowski","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cassandra M.","family":"Izzo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erin","family":"Horn","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Toloo","family":"Taghian","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paola","family":"Rodriguez","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ana Rita","family":"Batista","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lauren E.","family":"Ellis","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Misako","family":"Hwang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Douglas R.","family":"Martin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Heather","family":"Gray-Edwards","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miguel","family":"Sena-Esteves","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymthe.2020.06.021_bib1","doi-asserted-by":"crossref","first-page":"e1233","DOI":"10.1542\/peds.2011-0078","article-title":"Natural history of infantile G(M2) gangliosidosis","volume":"128","author":"Bley","year":"2011","journal-title":"Pediatrics"},{"key":"10.1016\/j.ymthe.2020.06.021_bib2","doi-asserted-by":"crossref","first-page":"10954","DOI":"10.1073\/pnas.97.20.10954","article-title":"Microglial activation precedes acute neurodegeneration in Sandhoff disease and is suppressed by bone marrow transplantation","volume":"97","author":"Wada","year":"2000","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2020.06.021_bib3","doi-asserted-by":"crossref","first-page":"974","DOI":"10.1093\/brain\/awg089","article-title":"Central nervous system inflammation is a hallmark of pathogenesis in mouse models of GM1 and GM2 gangliosidosis","volume":"126","author":"Jeyakumar","year":"2003","journal-title":"Brain"},{"key":"10.1016\/j.ymthe.2020.06.021_bib4","doi-asserted-by":"crossref","first-page":"102","DOI":"10.1016\/j.expneurol.2014.09.020","article-title":"Biomarkers for disease progression and AAV therapeutic efficacy in feline Sandhoff disease","volume":"263","author":"Bradbury","year":"2015","journal-title":"Exp. Neurol."},{"key":"10.1016\/j.ymthe.2020.06.021_bib5","doi-asserted-by":"crossref","first-page":"848","DOI":"10.1212\/WNL.29.6.848","article-title":"Enzyme replacement in Tay-Sachs disease","volume":"29","author":"von Specht","year":"1979","journal-title":"Neurology"},{"key":"10.1016\/j.ymthe.2020.06.021_bib6","doi-asserted-by":"crossref","first-page":"428","DOI":"10.1126\/science.276.5311.428","article-title":"Prevention of lysosomal storage in Tay-Sachs mice treated with N-butyldeoxynojirimycin","volume":"276","author":"Platt","year":"1997","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2020.06.021_bib7","doi-asserted-by":"crossref","first-page":"1125","DOI":"10.1016\/j.neuint.2007.12.001","article-title":"N-butyldeoxygalactonojirimycin reduces brain ganglioside and GM2 content in neonatal Sandhoff disease mice","volume":"52","author":"Baek","year":"2008","journal-title":"Neurochem. Int."},{"key":"10.1016\/j.ymthe.2020.06.021_bib8","doi-asserted-by":"crossref","first-page":"439","DOI":"10.1038\/nm1548","article-title":"Stem cells act through multiple mechanisms to benefit mice with neurodegenerative metabolic disease","volume":"13","author":"Lee","year":"2007","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2020.06.021_bib9","doi-asserted-by":"crossref","first-page":"1335","DOI":"10.1007\/s11064-012-0718-0","article-title":"Therapeutic effects of stem cells and substrate reduction in juvenile Sandhoff mice","volume":"37","author":"Arthur","year":"2012","journal-title":"Neurochem. Res."},{"key":"10.1016\/j.ymthe.2020.06.021_bib10","doi-asserted-by":"crossref","first-page":"642","DOI":"10.1002\/ana.20242","article-title":"NSAIDs increase survival in the Sandhoff disease mouse: synergy with N-butyldeoxynojirimycin","volume":"56","author":"Jeyakumar","year":"2004","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.ymthe.2020.06.021_bib11","doi-asserted-by":"crossref","first-page":"327","DOI":"10.1182\/blood.V97.1.327","article-title":"Enhanced survival in Sandhoff disease mice receiving a combination of substrate deprivation therapy and bone marrow transplantation","volume":"97","author":"Jeyakumar","year":"2001","journal-title":"Blood"},{"key":"10.1016\/j.ymthe.2020.06.021_bib12","doi-asserted-by":"crossref","first-page":"1028","DOI":"10.1002\/jnr.20798","article-title":"Influence of caloric restriction on motor behavior, longevity, and brain lipid composition in Sandhoff disease mice","volume":"83","author":"Denny","year":"2006","journal-title":"J.\u00a0Neurosci. Res."},{"key":"10.1016\/j.ymthe.2020.06.021_bib13","doi-asserted-by":"crossref","first-page":"1525","DOI":"10.1111\/j.1471-4159.2010.06733.x","article-title":"Restricted ketogenic diet enhances the therapeutic action of N-butyldeoxynojirimycin towards brain GM2 accumulation in adult Sandhoff disease mice","volume":"113","author":"Denny","year":"2010","journal-title":"J.\u00a0Neurochem."},{"key":"10.1016\/j.ymthe.2020.06.021_bib14","doi-asserted-by":"crossref","first-page":"425","DOI":"10.1097\/GIM.0b013e3181a1b5c5","article-title":"Miglustat in late-onset Tay-Sachs disease: a 12-month, randomized, controlled clinical study with 24\u00a0months of extended treatment","volume":"11","author":"Shapiro","year":"2009","journal-title":"Genet. Med."},{"key":"10.1016\/j.ymthe.2020.06.021_bib15","doi-asserted-by":"crossref","first-page":"10373","DOI":"10.1073\/pnas.0603765103","article-title":"Effective gene therapy in an authentic model of Tay-Sachs-related diseases","volume":"103","author":"Cach\u00f3n-Gonz\u00e1lez","year":"2006","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2020.06.021_bib16","doi-asserted-by":"crossref","first-page":"1489","DOI":"10.1038\/mt.2012.44","article-title":"Gene transfer corrects acute GM2 gangliosidosis\u2013potential therapeutic contribution of perivascular enzyme flow","volume":"20","author":"Cachon-Gonzalez","year":"2012","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2020.06.021_bib17","doi-asserted-by":"crossref","first-page":"730","DOI":"10.1093\/hmg\/ddt459","article-title":"Reversibility of neuropathology in Tay-Sachs-related diseases","volume":"23","author":"Cach\u00f3n-Gonz\u00e1lez","year":"2014","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2020.06.021_bib18","doi-asserted-by":"crossref","first-page":"181","DOI":"10.1038\/gt.2014.108","article-title":"Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease","volume":"22","author":"McCurdy","year":"2015","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2020.06.021_bib19","doi-asserted-by":"crossref","first-page":"7","DOI":"10.1177\/1759091415569908","article-title":"AAV-mediated gene delivery in a feline model of Sandhoff disease corrects lysosomal storage in the central nervous system","volume":"7","author":"Rockwell","year":"2015","journal-title":"ASN Neuro"},{"key":"10.1016\/j.ymthe.2020.06.021_bib20","doi-asserted-by":"crossref","first-page":"312","DOI":"10.1089\/hum.2017.163","article-title":"AAV gene therapy in a sheep model of Tay-Sachs disease","volume":"29","author":"Gray-Edwards","year":"2017","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2020.06.021_bib21","doi-asserted-by":"crossref","first-page":"414","DOI":"10.1038\/mt.2014.240","article-title":"Long-term correction of Sandhoff disease following intravenous delivery of rAAV9 to mouse neonates","volume":"23","author":"Walia","year":"2015","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2020.06.021_bib22","doi-asserted-by":"crossref","first-page":"954","DOI":"10.1093\/hmg\/ddy012","article-title":"Intravenous administration of scAAV9-Hexb normalizes lifespan and prevents pathology in Sandhoff disease mice","volume":"27","author":"Niemir","year":"2018","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2020.06.021_bib23","doi-asserted-by":"crossref","first-page":"170","DOI":"10.1038\/ng1095-170","article-title":"Mouse models of Tay-Sachs and Sandhoff diseases differ in neurologic phenotype and ganglioside metabolism","volume":"11","author":"Sango","year":"1995","journal-title":"Nat. Genet."},{"key":"10.1016\/j.ymthe.2020.06.021_bib25","doi-asserted-by":"crossref","first-page":"2636","DOI":"10.1128\/jvi.62.8.2636-2643.1988","article-title":"A segment of the 5\u2032 nontranslated region of encephalomyocarditis virus RNA directs internal entry of ribosomes during in\u00a0vitro translation","volume":"62","author":"Jang","year":"1988","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.ymthe.2020.06.021_bib26","doi-asserted-by":"crossref","first-page":"9488","DOI":"10.1073\/pnas.0603597103","article-title":"Ribosomal shunting mediated by a translational enhancer element that base pairs to 18S rRNA","volume":"103","author":"Chappell","year":"2006","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2020.06.021_bib27","doi-asserted-by":"crossref","first-page":"198","DOI":"10.1038\/sj.gt.3300811","article-title":"Use of the 2A sequence from foot-and-mouth disease virus in the generation of retroviral vectors for gene therapy","volume":"6","author":"de Felipe","year":"1999","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2020.06.021_bib28","doi-asserted-by":"crossref","first-page":"864","DOI":"10.1038\/sj.gt.3301469","article-title":"Recombinant AAV vectors containing the foot and mouth disease virus 2A sequence confer efficient bicistronic gene expression in cultured cells and rat substantia nigra neurons","volume":"8","author":"Furler","year":"2001","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2020.06.021_bib29","doi-asserted-by":"crossref","first-page":"6388","DOI":"10.1073\/pnas.96.11.6388","article-title":"Delayed symptom onset and increased life expectancy in Sandhoff disease mice treated with N-butyldeoxynojirimycin","volume":"96","author":"Jeyakumar","year":"1999","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2020.06.021_bib30","doi-asserted-by":"crossref","first-page":"204","DOI":"10.1038\/nbt.3440","article-title":"Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain","volume":"34","author":"Deverman","year":"2016","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2020.06.021_bib31","doi-asserted-by":"crossref","first-page":"510","DOI":"10.1089\/hum.2016.109","article-title":"Direct Intracranial Injection of AAVrh8 Encoding Monkey \u03b2-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain","volume":"28","author":"Golebiowski","year":"2017","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2020.06.021_bib32","doi-asserted-by":"crossref","first-page":"1306","DOI":"10.1038\/mt.2013.86","article-title":"Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy","volume":"21","author":"Bradbury","year":"2013","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2020.06.021_bib33","doi-asserted-by":"crossref","first-page":"101","DOI":"10.1016\/0009-9120(95)00003-R","article-title":"Beta-hexosaminidase: biosynthesis and processing of the normal enzyme, and identification of mutations causing Jewish Tay-Sachs disease","volume":"28","author":"Mahuran","year":"1995","journal-title":"Clin. Biochem."},{"key":"10.1016\/j.ymthe.2020.06.021_bib34","doi-asserted-by":"crossref","first-page":"105","DOI":"10.1016\/S0925-4439(99)00074-5","article-title":"Biochemical consequences of mutations causing the GM2 gangliosidoses","volume":"1455","author":"Mahuran","year":"1999","journal-title":"Biochim. Biophys. Acta"},{"key":"10.1016\/j.ymthe.2020.06.021_bib35","doi-asserted-by":"crossref","first-page":"1314","DOI":"10.1038\/gt.2009.106","article-title":"Delivering multiple gene products in the brain from a single adeno-associated virus vector","volume":"16","author":"Foti","year":"2009","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2020.06.021_bib36","doi-asserted-by":"crossref","first-page":"269","DOI":"10.1016\/0378-1119(89)90209-6","article-title":"Expression vector system based on the chicken beta-actin promoter directs efficient production of interleukin-5","volume":"79","author":"Miyazaki","year":"1989","journal-title":"Gene"},{"key":"10.1016\/j.ymthe.2020.06.021_bib37","first-page":"4046","article-title":"Housekeeping Na,K-ATPase alpha 1 subunit gene promoter is composed of multiple cis elements to which common and cell type-specific factors bind","volume":"12","author":"Suzuki-Yagawa","year":"1992","journal-title":"Mol. Cell. Biol."},{"key":"10.1016\/j.ymthe.2020.06.021_bib38","doi-asserted-by":"crossref","first-page":"10613","DOI":"10.1073\/pnas.90.22.10613","article-title":"Stable in\u00a0vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector","volume":"90","author":"Flotte","year":"1993","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2020.06.021_bib39","doi-asserted-by":"crossref","first-page":"59","DOI":"10.1038\/nbt.1515","article-title":"Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes","volume":"27","author":"Foust","year":"2009","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2020.06.021_bib40","doi-asserted-by":"crossref","first-page":"271","DOI":"10.1038\/nbt.1610","article-title":"Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN","volume":"28","author":"Foust","year":"2010","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2020.06.021_bib41","doi-asserted-by":"crossref","first-page":"1971","DOI":"10.1038\/mt.2011.157","article-title":"Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders","volume":"19","author":"Bevan","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2020.06.021_bib42","doi-asserted-by":"crossref","first-page":"1187","DOI":"10.1038\/mt.2009.71","article-title":"Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons","volume":"17","author":"Duque","year":"2009","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2020.06.021_bib43","doi-asserted-by":"crossref","first-page":"15057","DOI":"10.1038\/mtm.2015.57","article-title":"Construction of a hybrid \u03b2-hexosaminidase subunit capable of forming stable homodimers that hydrolyze GM2 ganglioside in\u00a0vivo","volume":"3","author":"Tropak","year":"2016","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2020.06.021_bib44","doi-asserted-by":"crossref","first-page":"509","DOI":"10.1089\/hum.2016.013","article-title":"Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease","volume":"27","author":"Karumuthil-Melethil","year":"2016","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2020.06.021_bib45","first-page":"105","article-title":"Induction of Immune Tolerance towards a Human Protein- HexM Expressed through AAV Gene Transfer in Sandhoff Mice","volume":"26","author":"Kot","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2020.06.021_bib46","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1016\/j.omtm.2018.10.011","article-title":"Efficacy of a Bicistronic Vector for Correction of Sandhoff Disease in a Mouse Model","volume":"12","author":"Woodley","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2020.06.021_bib47","doi-asserted-by":"crossref","first-page":"1338","DOI":"10.1177\/0271678X19865917","article-title":"Brain endothelial specific gene therapy improves experimental Sandhoff disease","volume":"40","author":"Dogbevia","year":"2019","journal-title":"J.\u00a0Cereb. Blood Flow Metab."},{"key":"10.1016\/j.ymthe.2020.06.021_bib48","doi-asserted-by":"crossref","first-page":"1172","DOI":"10.1038\/nn.4593","article-title":"Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems","volume":"20","author":"Chan","year":"2017","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.ymthe.2020.06.021_bib49","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.\u00a0Engl. J. Med."},{"key":"10.1016\/j.ymthe.2020.06.021_bib50","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.\u00a0Engl. J. Med."},{"key":"10.1016\/j.ymthe.2020.06.021_bib51","doi-asserted-by":"crossref","first-page":"2519","DOI":"10.1056\/NEJMoa1708483","article-title":"AAV5-Factor VIII Gene Transfer in Severe Hemophilia A","volume":"377","author":"Rangarajan","year":"2017","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.ymthe.2020.06.021_bib52","doi-asserted-by":"crossref","first-page":"208","DOI":"10.1089\/humc.2017.146","article-title":"Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease","volume":"28","author":"Corti","year":"2017","journal-title":"Hum. Gene Ther. Clin. Dev."},{"key":"10.1016\/j.ymthe.2020.06.021_bib24","doi-asserted-by":"crossref","first-page":"90","DOI":"10.1089\/hum.2019.186","article-title":"Ly6a Differential Expression in Blood-Brain Barrier Is Responsible for Strain Specific Central Nervous System Transduction Profile of AAV-PHP.B","volume":"31","author":"Batista","year":"2020","journal-title":"Hum. Gene Ther."}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001620303117?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001620303117?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,26]],"date-time":"2025-10-26T17:57:52Z","timestamp":1761501472000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001620303117"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,10]]},"references-count":52,"journal-issue":{"issue":"10","published-print":{"date-parts":[[2020,10]]}},"alternative-id":["S1525001620303117"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2020.06.021","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2020,10]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2020.06.021","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2020.09.017","name":"associatedlink","label":"CrossRef DOI link to the associated document"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2020 The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}]}}