{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,6]],"date-time":"2026-06-06T08:41:04Z","timestamp":1780735264881,"version":"3.54.1"},"reference-count":49,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2021,12,1]],"date-time":"2021-12-01T00:00:00Z","timestamp":1638316800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2021,12,1]],"date-time":"2021-12-01T00:00:00Z","timestamp":1638316800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2021,8,23]],"date-time":"2021-08-23T00:00:00Z","timestamp":1629676800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["T32 GM008382"],"award-info":[{"award-number":["T32 GM008382"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["10R01EB005866-08"],"award-info":[{"award-number":["10R01EB005866-08"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100007920","name":"University of Massachusetts Medical School","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007920","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100007059","name":"Northwestern University","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007059","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000001","name":"National Science Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000001","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy - Methods &amp; Clinical Development"],"published-print":{"date-parts":[[2021,12]]},"DOI":"10.1016\/j.omtm.2021.08.003","type":"journal-article","created":{"date-parts":[[2021,8,25]],"date-time":"2021-08-25T23:16:29Z","timestamp":1629933389000},"page":"128-134","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":17,"special_numbering":"C","title":["Real-time MR tracking of AAV gene therapy with \u03b2gal-responsive MR probe in a murine model of GM1-gangliosidosis"],"prefix":"10.1016","volume":"23","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7914-4733","authenticated-orcid":false,"given":"Toloo","family":"Taghian","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ana Rita","family":"Batista","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1156-1666","authenticated-orcid":false,"given":"Sarah","family":"Kamper","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Caldwell","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura","family":"Lilley","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5444-1168","authenticated-orcid":false,"given":"Hao","family":"Li","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paola","family":"Rodriguez","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katerina","family":"Mesa","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shaokuan","family":"Zheng","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5144-9110","authenticated-orcid":false,"given":"Robert M.","family":"King","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthew J.","family":"Gounis","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sophia","family":"Todeasa","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3037-9125","authenticated-orcid":false,"given":"Anne","family":"Maguire","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3444-8407","authenticated-orcid":false,"given":"Douglas R.","family":"Martin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miguel","family":"Sena-Esteves","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6202-1155","authenticated-orcid":false,"given":"Thomas J.","family":"Meade","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Heather L.","family":"Gray-Edwards","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2021.08.003_bib1","doi-asserted-by":"crossref","first-page":"1887","DOI":"10.1056\/NEJMoa1414221","article-title":"Long-term effect of gene therapy on Leber\u2019s congenital amaurosis","volume":"372","author":"Bainbridge","year":"2015","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2021.08.003_bib2","doi-asserted-by":"crossref","first-page":"1713","DOI":"10.1056\/NEJMoa1706198","article-title":"Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy","volume":"377","author":"Mendell","year":"2017","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2021.08.003_bib3","doi-asserted-by":"crossref","first-page":"2215","DOI":"10.1056\/NEJMoa1708538","article-title":"Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant","volume":"377","author":"George","year":"2017","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2021.08.003_bib4","doi-asserted-by":"crossref","first-page":"4906","DOI":"10.1172\/JCI133953","article-title":"Krabbe disease successfully treated via monotherapy of intrathecal gene therapy","volume":"130","author":"Bradbury","year":"2020","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2021.08.003_bib5","doi-asserted-by":"crossref","first-page":"206","DOI":"10.1038\/mt.2015.189","article-title":"Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII","volume":"24","author":"Gurda","year":"2016","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.08.003_bib6","doi-asserted-by":"crossref","first-page":"231ra48","DOI":"10.1126\/scitranslmed.3007733","article-title":"Sustained normalization of neurological disease after intracranial gene therapy in a feline model","volume":"6","author":"McCurdy","year":"2014","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtm.2021.08.003_bib7","doi-asserted-by":"crossref","first-page":"726","DOI":"10.1038\/mt.2015.231","article-title":"Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector","volume":"24","author":"Choudhury","year":"2016","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.08.003_bib8","doi-asserted-by":"crossref","first-page":"312","DOI":"10.1089\/hum.2017.163","article-title":"Adeno-Associated Virus Gene Therapy in a Sheep Model of Tay-Sachs Disease","volume":"29","author":"Gray-Edwards","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2021.08.003_bib9","doi-asserted-by":"crossref","first-page":"413","DOI":"10.1016\/j.ymthe.2005.04.018","article-title":"AAV2-mediated ocular gene therapy for infantile neuronal ceroid lipofuscinosis","volume":"12","author":"Griffey","year":"2005","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.08.003_bib10","doi-asserted-by":"crossref","first-page":"106","DOI":"10.1016\/j.ymthe.2004.03.018","article-title":"AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice","volume":"10","author":"Hennig","year":"2004","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.08.003_bib11","first-page":"52","article-title":"Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1","volume":"26","author":"Chandler","year":"2017","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtm.2021.08.003_bib12","doi-asserted-by":"crossref","first-page":"313ra180","DOI":"10.1126\/scitranslmed.aac6191","article-title":"AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease","volume":"7","author":"Katz","year":"2015","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtm.2021.08.003_bib13","doi-asserted-by":"crossref","first-page":"160","DOI":"10.1016\/j.omtm.2018.02.005","article-title":"Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy","volume":"9","author":"Bailey","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2021.08.003_bib14","first-page":"153","article-title":"Recent endeavors in MECP2 gene transfer for gene therapy of Rett syndrome","volume":"24","author":"Sinnett","year":"2017","journal-title":"Discov. Med."},{"key":"10.1016\/j.omtm.2021.08.003_bib15","doi-asserted-by":"crossref","first-page":"2958","DOI":"10.1038\/s41467-019-10809-6","article-title":"Bone-targeting AAV-mediated silencing of Schnurri-3 prevents bone loss in osteoporosis","volume":"10","author":"Yang","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2021.08.003_bib16","doi-asserted-by":"crossref","first-page":"429","DOI":"10.1007\/978-1-4939-3271-9_30","article-title":"Gene Therapy for the Treatment of Neurological Disorders: Metabolic Disorders","volume":"1382","author":"Gessler","year":"2016","journal-title":"Methods Mol. Biol."},{"key":"10.1016\/j.omtm.2021.08.003_bib17","doi-asserted-by":"crossref","first-page":"1239","DOI":"10.1089\/hum.2011.053","article-title":"Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing \u03b11-antitrypsin: interim results","volume":"22","author":"Flotte","year":"2011","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2021.08.003_bib18","doi-asserted-by":"crossref","first-page":"464","DOI":"10.1016\/j.ymthe.2020.12.007","article-title":"Current Clinical Applications of In\u00a0Vivo Gene Therapy with AAVs","volume":"29","author":"Mendell","year":"2021","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.08.003_bib19","doi-asserted-by":"crossref","first-page":"151","DOI":"10.1056\/NEJMoa2005056","article-title":"SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS","volume":"383","author":"Mueller","year":"2020","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2021.08.003_bib20","doi-asserted-by":"crossref","first-page":"957","DOI":"10.1089\/hum.2019.012","article-title":"Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys","volume":"30","author":"Hordeaux","year":"2019","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2021.08.003_bib21","unstructured":"ClinicalTrials.gov (2021). A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis. NCT04273269."},{"key":"10.1016\/j.omtm.2021.08.003_bib22","unstructured":"ClinicalTrials.gov (2021). A Phase 1\/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human \u2264-Galactosidase in Type II GM1 Gangliosidosis. NCT03952637."},{"key":"10.1016\/j.omtm.2021.08.003_bib23","unstructured":"ClinicalTrials.gov (2021). Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Subjects With GM1 Gangliosidosis (Imagine-1). NCT04713475."},{"key":"10.1016\/j.omtm.2021.08.003_bib24","unstructured":"ClinicalTrials.gov (2021). A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease. NCT04669535."},{"key":"10.1016\/j.omtm.2021.08.003_bib25","unstructured":"ClinicalTrials.gov (2021). A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101. NCT04655911."},{"key":"10.1016\/j.omtm.2021.08.003_bib26","unstructured":"ClinicalTrials.gov (2021). RGX-121 Gene Therapy in Patients With MPS II (Hunter Syndrome). NCT03566043."},{"key":"10.1016\/j.omtm.2021.08.003_bib27","unstructured":"ClinicalTrials.gov (2021). An Open-label, Phase 1\/2 Trial of Gene Therapy 4D-310 in Adult Males With Fabry Disease. NCT04519749."},{"key":"10.1016\/j.omtm.2021.08.003_bib28","unstructured":"ClinicalTrials.gov (2020). A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102. NCT04360265."},{"key":"10.1016\/j.omtm.2021.08.003_bib29","unstructured":"ClinicalTrials.gov (2021). Intrathecal Administration of scAAV9\/JeT-GAN for the Treatment of Giant Axonal Neuropathy. NCT02362438."},{"key":"10.1016\/j.omtm.2021.08.003_bib30","unstructured":"ClinicalTrials.gov (2021). A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE). NCT04093349."},{"key":"10.1016\/j.omtm.2021.08.003_bib31","first-page":"951","article-title":"Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review","volume":"260","author":"Toscano","year":"2013","journal-title":"J.\u00a0Neurol."},{"key":"10.1016\/j.omtm.2021.08.003_bib32","doi-asserted-by":"crossref","first-page":"388","DOI":"10.1002\/anie.201909933","article-title":"Self-Immolative Activation of \u03b2-Galactosidase-Responsive Probes for In\u00a0Vivo MR Imaging in Mouse Models","volume":"59","author":"Lilley","year":"2020","journal-title":"Angew. Chem. Int. Ed. Engl."},{"key":"10.1016\/j.omtm.2021.08.003_bib33","doi-asserted-by":"crossref","first-page":"957","DOI":"10.1021\/acs.chemrev.8b00363","article-title":"Chemistry of MRI Contrast Agents: Current Challenges and New Frontiers","volume":"119","author":"Wahsner","year":"2019","journal-title":"Chem. Rev."},{"key":"10.1016\/j.omtm.2021.08.003_bib34","doi-asserted-by":"crossref","first-page":"4353","DOI":"10.1093\/hmg\/ddv168","article-title":"Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan","volume":"24","author":"Weismann","year":"2015","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtm.2021.08.003_bib35","doi-asserted-by":"crossref","first-page":"e13468","DOI":"10.1371\/journal.pone.0013468","article-title":"AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival","volume":"5","author":"Baek","year":"2010","journal-title":"PLoS ONE"},{"key":"10.1016\/j.omtm.2021.08.003_bib36","doi-asserted-by":"crossref","first-page":"205","DOI":"10.1093\/hmg\/6.2.205","article-title":"Generalized CNS disease and massive GM1-ganglioside accumulation in mice defective in lysosomal acid beta-galactosidase","volume":"6","author":"Hahn","year":"1997","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtm.2021.08.003_bib37","doi-asserted-by":"crossref","first-page":"411","DOI":"10.1016\/j.ymthe.2019.11.012","article-title":"A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna","volume":"28","author":"Taghian","year":"2020","journal-title":"Mol. Ther."},{"issue":"Suppl 2","key":"10.1016\/j.omtm.2021.08.003_bib38","doi-asserted-by":"crossref","first-page":"120","DOI":"10.1186\/s13052-018-0562-1","article-title":"Enzyme replacement therapy: efficacy and limitations","volume":"44","author":"Concolino","year":"2018","journal-title":"Ital. J. Pediatr."},{"key":"10.1016\/j.omtm.2021.08.003_bib39","doi-asserted-by":"crossref","first-page":"291","DOI":"10.21037\/atm.2019.04.15","article-title":"Challenges in treating Pompe disease: an industry perspective","volume":"7","author":"Do","year":"2019","journal-title":"Ann. Transl. Med."},{"key":"10.1016\/j.omtm.2021.08.003_bib40","first-page":"58","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.2021.08.003_bib41","doi-asserted-by":"crossref","first-page":"396","DOI":"10.1097\/RLI.0000000000000352","article-title":"Quantification and Assessment of the Chemical Form of Residual Gadolinium in the Brain After Repeated Administration of Gadolinium-Based Contrast Agents: Comparative Study in Rats","volume":"52","author":"Frenzel","year":"2017","journal-title":"Invest. Radiol."},{"key":"10.1016\/j.omtm.2021.08.003_bib42","first-page":"1","volume":"10.3","year":"2017"},{"key":"10.1016\/j.omtm.2021.08.003_bib43","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1177\/2054358118778573","article-title":"Gadolinium-Based Contrast Agents in Kidney Disease: A Comprehensive Review and Clinical Practice Guideline Issued by the Canadian Association of Radiologists","volume":"5","author":"Schieda","year":"2018","journal-title":"Can. J. Kidney Health Dis."},{"key":"10.1016\/j.omtm.2021.08.003_bib44","doi-asserted-by":"crossref","first-page":"109","DOI":"10.1097\/RLI.0000000000000000","article-title":"Incidence of nephrogenic systemic fibrosis in patients undergoing dialysis after contrast-enhanced magnetic resonance imaging with gadolinium-based contrast agents: the Prospective Fibrose Nephrog\u00e9nique Syst\u00e9mique study","volume":"49","author":"Amet","year":"2014","journal-title":"Invest. Radiol."},{"key":"10.1016\/j.omtm.2021.08.003_bib45","doi-asserted-by":"crossref","first-page":"55","DOI":"10.1097\/RLI.0000000000000307","article-title":"Gadobutrol in Renally Impaired Patients: Results of the GRIP Study","volume":"52","author":"Michaely","year":"2017","journal-title":"Invest. Radiol."},{"key":"10.1016\/j.omtm.2021.08.003_bib46","doi-asserted-by":"crossref","first-page":"1927","DOI":"10.1007\/s00467-013-2636-z","article-title":"Gadolinium and nephrogenic systemic fibrosis: an update","volume":"29","author":"Weller","year":"2014","journal-title":"Pediatr. Nephrol."},{"key":"10.1016\/j.omtm.2021.08.003_bib47","doi-asserted-by":"crossref","first-page":"W138","DOI":"10.2214\/AJR.10.4885","article-title":"Incidence of immediate gadolinium contrast media reactions","volume":"196","author":"Prince","year":"2011","journal-title":"AJR Am. J. Roentgenol."},{"key":"10.1016\/j.omtm.2021.08.003_bib48","doi-asserted-by":"crossref","first-page":"12847","DOI":"10.1021\/ja042162r","article-title":"A gadolinium chelate for detection of beta-glucuronidase: a self-immolative approach","volume":"127","author":"Duimstra","year":"2005","journal-title":"J.\u00a0Am. Chem. Soc."},{"key":"10.1016\/j.omtm.2021.08.003_bib49","doi-asserted-by":"crossref","DOI":"10.1101\/pdb.prot095539","article-title":"Release of the Cloned Recombinant Adenovirus Genome for Rescue and Expansion","volume":"2019","author":"Su","year":"2019","journal-title":"Cold Spring Harb. Protoc."}],"container-title":["Molecular Therapy - Methods &amp; Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050121001297?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050121001297?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,5,15]],"date-time":"2026-05-15T17:54:54Z","timestamp":1778867694000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050121001297"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,12]]},"references-count":49,"alternative-id":["S2329050121001297"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2021.08.003","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2021,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Real-time MR tracking of AAV gene therapy with \u03b2gal-responsive MR probe in a murine model of GM1-gangliosidosis","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2021.08.003","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"name":"copyright","label":"Copyright"}]}}