{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,10]],"date-time":"2026-09-10T06:11:35Z","timestamp":1789020695256,"version":"build-2803163510"},"reference-count":61,"publisher":"Elsevier BV","issue":"1","license":[{"start":{"date-parts":[[2024,3,1]],"date-time":"2024-03-01T00:00:00Z","timestamp":1709251200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2024,3,1]],"date-time":"2024-03-01T00:00:00Z","timestamp":1709251200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2023,12,1]],"date-time":"2023-12-01T00:00:00Z","timestamp":1701388800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100009056","name":"GlycoNet","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100009056","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100012515","name":"Cyrus Tang Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100012515","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy - Methods & Clinical Development"],"published-print":{"date-parts":[[2024,3]]},"DOI":"10.1016\/j.omtm.2023.101168","type":"journal-article","created":{"date-parts":[[2023,12,5]],"date-time":"2023-12-05T09:04:25Z","timestamp":1701767065000},"page":"101168","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":10,"title":["Intrathecal delivery of a bicistronic AAV9 vector expressing \u03b2-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study"],"prefix":"10.1016","volume":"32","author":[{"ORCID":"https:\/\/orcid.org\/0009-0001-5971-8629","authenticated-orcid":false,"given":"Alex E.","family":"Ryckman","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Natalie M.","family":"Deschenes","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brianna M.","family":"Quinville","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karlaina J.L.","family":"Osmon","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melissa","family":"Mitchell","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhilin","family":"Chen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Steven J.","family":"Gray","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jagdeep S.","family":"Walia","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2023.101168_bib1","doi-asserted-by":"crossref","first-page":"58","DOI":"10.1159\/000112332","article-title":"Partial enzyme deficiencies: residual activities and the development of neurological disorders","volume":"6","author":"Conzelmann","year":"1983","journal-title":"Dev. Neurosci."},{"key":"10.1016\/j.omtm.2023.101168_bib2","doi-asserted-by":"crossref","first-page":"506","DOI":"10.1016\/j.nbd.2004.04.012","article-title":"Improved outcome of N-butyldeoxygalactonojirimycin-mediated substrate reduction therapy in a mouse model of Sandhoff disease","volume":"16","author":"Andersson","year":"2004","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.omtm.2023.101168_bib3","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.omtm.2023.101168_bib4","doi-asserted-by":"crossref","first-page":"159","DOI":"10.1007\/s12035-013-8605-5","article-title":"Chaperone therapy for GM2 gangliosidosis: effects of pyrimethamine on \u03b2-hexosaminidase activity in Sandhoff fibroblasts","volume":"50","author":"Chiricozzi","year":"2014","journal-title":"Mol. Neurobiol."},{"key":"10.1016\/j.omtm.2023.101168_bib5","doi-asserted-by":"crossref","first-page":"9150","DOI":"10.1074\/jbc.M609304200","article-title":"pyrimethamine as a potential pharmacological chaperone for late-onset forms of GM2 gangliosidosis","volume":"282","author":"Maegawa","year":"2007","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2023.101168_bib6","doi-asserted-by":"crossref","first-page":"1519","DOI":"10.1038\/mt.2010.113","article-title":"Introduction of an N-glycan sequon into HEXA enhances human beta-hexosaminidase cellular uptake in a model of Sandhoff disease","volume":"18","author":"Matsuoka","year":"2010","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2023.101168_bib7","doi-asserted-by":"crossref","first-page":"691","DOI":"10.1002\/ana.22262","article-title":"Highly phosphomannosylated enzyme replacement therapy for GM2 gangliosidosis","volume":"69","author":"Tsuji","year":"2011","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.omtm.2023.101168_bib8","doi-asserted-by":"crossref","first-page":"6","DOI":"10.1016\/j.ymgme.2010.09.004","article-title":"An open-label Phase I\/II clinical trial of pyrimethamine for the treatment of patients affected with chronic GM2 gangliosidosis (Tay\u2013Sachs or Sandhoff variants)","volume":"102","author":"Clarke","year":"2011","journal-title":"Mol. Genet. Metabol."},{"key":"10.1016\/j.omtm.2023.101168_bib9","doi-asserted-by":"crossref","first-page":"925","DOI":"10.1038\/sj.bmt.1705155","article-title":"Allogeneic BMT followed by substrate reduction therapy in a child with subacute Tay-Sachs disease","volume":"36","author":"Jacobs","year":"2005","journal-title":"Bone Marrow Transplant."},{"key":"10.1016\/j.omtm.2023.101168_bib10","doi-asserted-by":"crossref","first-page":"284","DOI":"10.1016\/j.ymgme.2009.04.013","article-title":"Pharmacokinetics, safety and tolerability of miglustat in the treatment of pediatric patients with GM2 gangliosidosis","volume":"97","author":"Maegawa","year":"2009","journal-title":"Mol. Genet. Metabol."},{"key":"10.1016\/j.omtm.2023.101168_bib11","doi-asserted-by":"crossref","first-page":"215","DOI":"10.1016\/j.ymgme.2009.06.005","article-title":"Substrate reduction therapy in juvenile GM2 gangliosidosis","volume":"98","author":"Maegawa","year":"2009","journal-title":"Mol. Genet. Metabol."},{"key":"10.1016\/j.omtm.2023.101168_bib12","doi-asserted-by":"crossref","first-page":"45","DOI":"10.1186\/s13023-015-0260-7","article-title":"Effect of cyclic, low dose pyrimethamine treatment in patients with Late Onset Tay Sachs: an open label, extended pilot study","volume":"10","author":"Osher","year":"2015","journal-title":"Orphanet J. Rare Dis."},{"key":"10.1016\/j.omtm.2023.101168_bib13","doi-asserted-by":"crossref","first-page":"2979","DOI":"10.1182\/blood-2008-03-140830","article-title":"Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes","volume":"112","author":"Prasad","year":"2008","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2023.101168_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.omtm.2023.101168_bib15","doi-asserted-by":"crossref","first-page":"583","DOI":"10.1016\/j.nbd.2005.04.017","article-title":"Bicistronic lentiviral vector corrects \u03b2-hexosaminidase deficiency in transduced and cross-corrected human Sandhoff fibroblasts","volume":"20","author":"Arfi","year":"2005","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.omtm.2023.101168_bib16","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.omtm.2023.101168_bib17","doi-asserted-by":"crossref","first-page":"831","DOI":"10.1093\/hmg\/8.5.831","article-title":"Adenoviral Gene Therapy of the Tay-Sachs Disease in Hexosaminidase A-Deficient Knock-Out Mice","volume":"8","author":"Guidotti","year":"1999","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtm.2023.101168_bib18","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.omtm.2023.101168_bib19","doi-asserted-by":"crossref","first-page":"2113","DOI":"10.1093\/hmg\/ddi216","article-title":"A direct gene transfer strategy via brain internal capsule reverses the biochemical defect in Tay\u2013Sachs disease","volume":"14","author":"Martino","year":"2005","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtm.2023.101168_bib20","doi-asserted-by":"crossref","DOI":"10.1016\/j.nbd.2019.104667","article-title":"Novel bicistronic lentiviral vectors correct \u03b2-Hexosaminidase deficiency in neural and hematopoietic stem cells and progeny: implications for in\u00a0vivo and ex\u00a0vivo gene therapy of GM2 gangliosidosis","volume":"134","author":"Ornaghi","year":"2020","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.omtm.2023.101168_bib21","doi-asserted-by":"crossref","first-page":"497","DOI":"10.1089\/hum.2016.015","article-title":"Systemic Gene Transfer of a Hexosaminidase Variant Using an scAAV9.47 Vector Corrects GM2 Gangliosidosis in Sandhoff Mice","volume":"27","author":"Osmon","year":"2016","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2023.101168_bib22","doi-asserted-by":"crossref","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.omtm.2023.101168_bib23","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":"2019","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2023.101168_bib24","first-page":"1","article-title":"Viral vectors: a look back and ahead on gene transfer technology","volume":"36","author":"Vannucci","year":"2013","journal-title":"New Microbiol."},{"key":"10.1016\/j.omtm.2023.101168_bib25","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1038\/s41591-021-01664-4","article-title":"AAV gene therapy for Tay-Sachs disease","volume":"28","author":"Flotte","year":"2022","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2023.101168_bib26","doi-asserted-by":"crossref","first-page":"2193","DOI":"10.1038\/s41598-017-02460-2","article-title":"Systematic comparison of 2A peptides for cloning multi-genes in a polycistronic vector","volume":"7","author":"Liu","year":"2017","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtm.2023.101168_bib27","doi-asserted-by":"crossref","first-page":"8","DOI":"10.1016\/j.mod.2015.07.007","article-title":"Formation and maintenance of the BBB","volume":"138","author":"Blanchette","year":"2015","journal-title":"Mech. Dev."},{"key":"10.1016\/j.omtm.2023.101168_bib28","doi-asserted-by":"crossref","first-page":"404","DOI":"10.3389\/fnins.2014.00404","article-title":"The rights and wrongs of blood-brain barrier permeability studies: a walk through 100 years of history","volume":"8","author":"Saunders","year":"2014","journal-title":"Front. Neurosci."},{"key":"10.1016\/j.omtm.2023.101168_bib29","doi-asserted-by":"crossref","first-page":"355ra118","DOI":"10.1126\/scitranslmed.aad9823","article-title":"Heat Shock Protein-based therapy as a potential candidate for treating sphingolipidoses","volume":"8","author":"Kirkegaard","year":"2016","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtm.2023.101168_bib30","doi-asserted-by":"crossref","first-page":"15","DOI":"10.1089\/hum.2017.026","article-title":"Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals","volume":"29","author":"Hinderer","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2023.101168_bib31","doi-asserted-by":"crossref","first-page":"478","DOI":"10.1089\/hum.2016.087","article-title":"Adeno-Associated Virus-Based Gene Therapy for CNS Diseases","volume":"27","author":"Hocquemiller","year":"2016","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2023.101168_bib32","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.omtm.2023.101168_bib33","doi-asserted-by":"crossref","first-page":"13478","DOI":"10.1074\/jbc.M308523200","article-title":"Pharmacological Enhancement of \u03b2-Hexosaminidase Activity in Fibroblasts from Adult Tay-Sachs and Sandhoff Patients","volume":"279","author":"Tropak","year":"2004","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2023.101168_bib34","doi-asserted-by":"crossref","first-page":"153","DOI":"10.1016\/j.chembiol.2006.12.006","article-title":"High-Throughput Screening for Human Lysosomal \u03b2-N-Acetyl Hexosaminidase Inhibitors Acting as Pharmacological Chaperones","volume":"14","author":"Tropak","year":"2007","journal-title":"Chem. Biol."},{"key":"10.1016\/j.omtm.2023.101168_bib35","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.omtm.2023.101168_bib36","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.omtm.2023.101168_bib37","doi-asserted-by":"crossref","first-page":"e164575","DOI":"10.1172\/JCI164575","article-title":"Intrathecal AAV9\/AP4M1 gene therapy for hereditary spastic paraplegia 50 shows safety and efficacy in preclinical studies","volume":"133","author":"Chen","year":"2023","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2023.101168_bib38","doi-asserted-by":"crossref","first-page":"989","DOI":"10.1016\/j.ymthe.2020.11.012","article-title":"Pre-clinical Gene Therapy with AAV9\/AGA in Aspartylglucosaminuria Mice Provides Evidence for Clinical Translation","volume":"29","author":"Chen","year":"2021","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2023.101168_bib39","doi-asserted-by":"crossref","DOI":"10.1172\/JCI146286","article-title":"AAV9\/MFSD8 gene therapy is effective in preclinical models of neuronal ceroid lipofuscinosis type 7 disease","volume":"132","author":"Chen","year":"2022","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2023.101168_bib40","doi-asserted-by":"crossref","first-page":"94","DOI":"10.1089\/hum.2022.163","article-title":"Biodistribution of Adeno-Associated Virus Gene Therapy Following Cerebrospinal Fluid-Directed Administration","volume":"34","author":"Chen","year":"2023","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2023.101168_bib41","doi-asserted-by":"crossref","first-page":"6751","DOI":"10.3390\/ijms22136751","article-title":"Investigating Immune Responses to the scAAV9-HEXM Gene Therapy Treatment in Tay-Sachs Disease and Sandhoff Disease Mouse Models","volume":"22","author":"Kot","year":"2021","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.omtm.2023.101168_bib42","doi-asserted-by":"crossref","first-page":"132","DOI":"10.3389\/fnmol.2019.00132","article-title":"Neuronal Cells Rearrangement During Aging and Neurodegenerative Disease: Metabolism, Oxidative Stress and Organelles Dynamic","volume":"12","author":"Castelli","year":"2019","journal-title":"Front. Mol. Neurosci."},{"key":"10.1016\/j.omtm.2023.101168_bib43","doi-asserted-by":"crossref","first-page":"E6881","DOI":"10.3390\/ijms21186881","article-title":"Metabolism of Glycosphingolipids and Their Role in the Pathophysiology of Lysosomal Storage Disorders","volume":"21","author":"Ryckman","year":"2020","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.omtm.2023.101168_bib44","doi-asserted-by":"crossref","first-page":"663","DOI":"10.1177\/0300985813501334","article-title":"Neoplastic and Nonneoplastic Lesions in Aging Mice of Unique and Common Inbred Strains Contribution to Modeling of Human Neoplastic Diseases","volume":"51","author":"Szymanska","year":"2014","journal-title":"Vet. Pathol."},{"key":"10.1016\/j.omtm.2023.101168_bib45","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.omtm.2023.101168_bib46","doi-asserted-by":"crossref","first-page":"477","DOI":"10.1126\/science.1142658","article-title":"AAV vector integration sites in mouse hepatocellular carcinoma","volume":"317","author":"Donsante","year":"2007","journal-title":"Science"},{"key":"10.1016\/j.omtm.2023.101168_bib47","doi-asserted-by":"crossref","first-page":"299","DOI":"10.1016\/j.ymthe.2005.03.020","article-title":"No evidence for tumorigenesis of AAV vectors in a large-scale study in mice","volume":"12","author":"Bell","year":"2005","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2023.101168_bib48","doi-asserted-by":"crossref","first-page":"3311","DOI":"10.1182\/blood-2010-08-302729","article-title":"Assessing the potential for AAV vector genotoxicity in a murine model","volume":"117","author":"Li","year":"2011","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2023.101168_bib49","doi-asserted-by":"crossref","first-page":"122","DOI":"10.4103\/1673-5374.314310","article-title":"Functionality of a bicistronic construction containing HEXA and HEXB genes encoding \u03b2-hexosaminidase A for cell-mediated therapy of GM2 gangliosidoses","volume":"17","author":"Shaimardanova","year":"2022","journal-title":"Neural Regen. Res."},{"key":"10.1016\/j.omtm.2023.101168_bib50","doi-asserted-by":"crossref","first-page":"262","DOI":"10.2174\/1566523221666210916153051","article-title":"Treatment of GM2 Gangliosidosis in Adult Sandhoff Mice Using an Intravenous Self-Complementary Hexosaminidase Vector","volume":"22","author":"Osmon","year":"2022","journal-title":"Curr. Gene Ther."},{"key":"10.1016\/j.omtm.2023.101168_bib51","first-page":"805","article-title":"Evaluation of Mice Undergoing Serial Oral Gavage While Awake or Anesthetized","volume":"55","author":"Jones","year":"2016","journal-title":"J.\u00a0Am. Assoc. Lab. Anim. Sci."},{"key":"10.1016\/j.omtm.2023.101168_bib52","doi-asserted-by":"crossref","first-page":"129","DOI":"10.1038\/laban0512-129","article-title":"The effects of repeated oral gavage on the health of male CD-1 mice","volume":"41","author":"Arantes-Rodrigues","year":"2012","journal-title":"Lab. Anim"},{"key":"10.1016\/j.omtm.2023.101168_bib53","doi-asserted-by":"crossref","first-page":"65","DOI":"10.1016\/j.taap.2012.01.025","article-title":"A less stressful alternative to oral gavage for pharmacological and toxicological studies in mice","volume":"260","author":"Walker","year":"2012","journal-title":"Toxicol. Appl. Pharmacol."},{"key":"10.1016\/j.omtm.2023.101168_bib54","doi-asserted-by":"crossref","first-page":"11654","DOI":"10.1523\/JNEUROSCI.4527-15.2016","article-title":"A Drosophila Model of Neuronopathic Gaucher Disease Demonstrates Lysosomal-Autophagic Defects and Altered mTOR Signalling and Is Functionally Rescued by Rapamycin","volume":"36","author":"Kinghorn","year":"2016","journal-title":"J.\u00a0Neurosci."},{"key":"10.1016\/j.omtm.2023.101168_bib55","doi-asserted-by":"crossref","first-page":"6926","DOI":"10.1523\/JNEUROSCI.0800-08.2008","article-title":"Autophagy Induction and Autophagosome Clearance in Neurons: Relationship to Autophagic Pathology in Alzheimer\u2019s Disease","volume":"28","author":"Boland","year":"2008","journal-title":"J.\u00a0Neurosci."},{"key":"10.1016\/j.omtm.2023.101168_bib56","doi-asserted-by":"crossref","DOI":"10.1016\/j.brainres.2020.146832","article-title":"Comparison of high-dose intracisterna magna and lumbar puncture intrathecal delivery of AAV9 in mice to treat neuropathies","volume":"1739","author":"Bailey","year":"2020","journal-title":"Brain Res."},{"key":"10.1016\/j.omtm.2023.101168_bib57","doi-asserted-by":"crossref","first-page":"450","DOI":"10.1038\/gt.2012.101","article-title":"Global CNS Gene Delivery and Evasion of Anti-AAV Neutralizing Antibodies by Intrathecal AAV Administration in Non-Human Primates","volume":"20","author":"Gray","year":"2013","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2023.101168_bib58","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1093\/hmg\/5.1.1","article-title":"Dramatically Different Phenotypes in Mouse Models of Human Tay-Sachs and Sandhoff Diseases","volume":"5","author":"Phaneuf","year":"1996","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtm.2023.101168_bib59","article-title":"Battery of Behavioral Tests Assessing General Locomotion, Muscular Strength, and Coordination in Mice","volume":"131","author":"Osmon","year":"2018","journal-title":"J.\u00a0Vis. Exp."},{"key":"10.1016\/j.omtm.2023.101168_bib60","doi-asserted-by":"crossref","first-page":"378","DOI":"10.1042\/bj0860378","article-title":"Detection and resolution of gangliosides in lipid extracts by thin-layer chromatography","volume":"86","author":"Wherrett","year":"1963","journal-title":"Biochem. J."},{"key":"10.1016\/j.omtm.2023.101168_bib61","doi-asserted-by":"crossref","first-page":"497","DOI":"10.1016\/S0021-9258(18)64849-5","article-title":"A simple method for the isolation and purification of total lipides from animal tissues","volume":"226","author":"Folch","year":"1957","journal-title":"J.\u00a0Biol. Chem."}],"container-title":["Molecular Therapy - Methods & Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050123002073?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050123002073?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2024,7,27]],"date-time":"2024-07-27T17:12:57Z","timestamp":1722100377000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050123002073"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,3]]},"references-count":61,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2024,3]]}},"alternative-id":["S2329050123002073"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2023.101168","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2024,3]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Intrathecal delivery of a bicistronic AAV9 vector expressing \u03b2-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2023.101168","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2023 The Authors.","name":"copyright","label":"Copyright"}],"article-number":"101168"}}