{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,24]],"date-time":"2026-09-24T18:17:24Z","timestamp":1790273844528,"version":"4.1.0"},"reference-count":57,"publisher":"Elsevier BV","issue":"3","license":[{"start":{"date-parts":[[2026,3,1]],"date-time":"2026-03-01T00:00:00Z","timestamp":1772323200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,3,1]],"date-time":"2026-03-01T00:00:00Z","timestamp":1772323200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,12,8]],"date-time":"2025-12-08T00:00:00Z","timestamp":1765152000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100004339","name":"Sanofi-Aventis US LLC","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004339","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":[[2026,3]]},"DOI":"10.1016\/j.ymthe.2025.12.011","type":"journal-article","created":{"date-parts":[[2025,12,9]],"date-time":"2025-12-09T16:53:45Z","timestamp":1765299225000},"page":"1707-1728","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":3,"title":["AAV gene therapy for GBA1-related diseases"],"prefix":"10.1016","volume":"34","author":[{"given":"Swathi","family":"Ayloo","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jae Cheon","family":"Ryu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shih-Ching","family":"Chou","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lydia","family":"Blatnik","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erik","family":"Wischhof","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jie","family":"Bu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lilu","family":"Guo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mahmud","family":"Hossain","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dhiman","family":"Ghosh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"William","family":"McCarty","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ann","family":"Byrne","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lilly","family":"Chai","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer","family":"Clarke","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Can","family":"Kayatekin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bailin","family":"Zhang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"S. Pablo","family":"Sardi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Martin","family":"Goulet","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bradford","family":"Elmer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3589-8749","authenticated-orcid":false,"given":"Christian","family":"Mueller","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shyam","family":"Ramachandran","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymthe.2025.12.011_bib1","doi-asserted-by":"crossref","DOI":"10.3390\/jcm9030736","article-title":"Glucocerebrosidase: Functions in and Beyond the Lysosome","volume":"9","author":"Boer","year":"2020","journal-title":"J. Clin. Med."},{"key":"10.1016\/j.ymthe.2025.12.011_bib2","doi-asserted-by":"crossref","first-page":"59","DOI":"10.1016\/j.ymgme.2020.12.291","article-title":"Gaucher disease: Basic and translational science needs for more complete therapy and management","volume":"132","author":"Grabowski","year":"2021","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2025.12.011_bib3","doi-asserted-by":"crossref","first-page":"104","DOI":"10.1016\/S1096-7192(03)00071-4","article-title":"Gaucher disease with parkinsonian manifestations: does glucocerebrosidase deficiency contribute to a vulnerability to parkinsonism?","volume":"79","author":"Tayebi","year":"2003","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2025.12.011_bib4","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1212\/01.WNL.0000072482.70963.D7","article-title":"Gaucher's disease with Parkinson's disease: clinical and pathological aspects","volume":"61","author":"Bembi","year":"2003","journal-title":"Neurology"},{"key":"10.1016\/j.ymthe.2025.12.011_bib5","doi-asserted-by":"crossref","first-page":"163","DOI":"10.1016\/j.neulet.2006.05.032","article-title":"Glucocerebrosidase mutations are not found in association with LRRK2 G2019S in subjects with parkinsonism","volume":"404","author":"Eblan","year":"2006","journal-title":"Neurosci. Lett."},{"key":"10.1016\/j.ymthe.2025.12.011_bib6","doi-asserted-by":"crossref","first-page":"908","DOI":"10.1212\/01.wnl.0000230215.41296.18","article-title":"Glucocerebrosidase mutations are an important risk factor for Lewy body disorders","volume":"67","author":"Goker-Alpan","year":"2006","journal-title":"Neurology"},{"key":"10.1016\/j.ymthe.2025.12.011_bib7","doi-asserted-by":"crossref","first-page":"195","DOI":"10.1016\/j.ymgme.2007.03.004","article-title":"Glucocerebrosidase mutations in Chinese subjects from Taiwan with sporadic Parkinson disease","volume":"91","author":"Ziegler","year":"2007","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2025.12.011_bib8","doi-asserted-by":"crossref","first-page":"1651","DOI":"10.1056\/NEJMoa0901281","article-title":"Multicenter analysis of glucocerebrosidase mutations in Parkinson's disease","volume":"361","author":"Sidransky","year":"2009","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.ymthe.2025.12.011_bib9","doi-asserted-by":"crossref","first-page":"674","DOI":"10.1002\/ana.24781","article-title":"Specifically neuropathic Gaucher's mutations accelerate cognitive decline in Parkinson's","volume":"80","author":"Liu","year":"2016","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.ymthe.2025.12.011_bib10","doi-asserted-by":"crossref","first-page":"662","DOI":"10.1002\/ana.24777","article-title":"Survival and dementia in GBA-associated Parkinson's disease: The mutation matters","volume":"80","author":"Cilia","year":"2016","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.ymthe.2025.12.011_bib11","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1002\/ana.26636","article-title":"Differences in Survival across Monogenic Forms of Parkinson's Disease","volume":"94","author":"Lanore","year":"2023","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.ymthe.2025.12.011_bib12","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1038\/nrneurol.2012.242","article-title":"100 years of Lewy pathology","volume":"9","author":"Goedert","year":"2013","journal-title":"Nat. Rev. Neurol."},{"key":"10.1016\/j.ymthe.2025.12.011_bib13","doi-asserted-by":"crossref","first-page":"407","DOI":"10.1016\/S1474-4422(23)00109-6","article-title":"Assessment of heterogeneity among participants in the Parkinson's Progression Markers Initiative cohort using alpha-synuclein seed amplification: a cross-sectional study","volume":"22","author":"Siderowf","year":"2023","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.ymthe.2025.12.011_bib14","doi-asserted-by":"crossref","first-page":"455","DOI":"10.1002\/ana.23614","article-title":"Glucocerebrosidase deficiency in substantia nigra of parkinson disease brains","volume":"72","author":"Gegg","year":"2012","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.ymthe.2025.12.011_bib15","doi-asserted-by":"crossref","first-page":"1350","DOI":"10.4161\/auto.29074","article-title":"Glucocerebrosidase deficits in sporadic Parkinson disease","volume":"10","author":"Murphy","year":"2014","journal-title":"Autophagy"},{"key":"10.1016\/j.ymthe.2025.12.011_bib16","doi-asserted-by":"crossref","first-page":"37","DOI":"10.1016\/j.cell.2011.06.001","article-title":"Gaucher disease glucocerebrosidase and alpha-synuclein form a bidirectional pathogenic loop in synucleinopathies","volume":"146","author":"Mazzulli","year":"2011","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2025.12.011_bib17","doi-asserted-by":"crossref","first-page":"6675","DOI":"10.1093\/hmg\/ddv372","article-title":"Glucocerebrosidase deficiency accelerates the accumulation of proteinase K-resistant alpha-synuclein and aggravates neurodegeneration in a Drosophila model of Parkinson's disease","volume":"24","author":"Suzuki","year":"2015","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2025.12.011_bib18","doi-asserted-by":"crossref","first-page":"9617","DOI":"10.1523\/JNEUROSCI.1525-17.2017","article-title":"Glucosylsphingosine Promotes alpha-Synuclein Pathology in Mutant GBA-Associated Parkinson's Disease","volume":"37","author":"Taguchi","year":"2017","journal-title":"J. Neurosci."},{"key":"10.1016\/j.ymthe.2025.12.011_bib19","doi-asserted-by":"crossref","first-page":"1216","DOI":"10.1002\/mds.28472","article-title":"The Mutation Matters: CSF Profiles of GCase, Sphingolipids, alpha-Synuclein in PD(GBA)","volume":"36","author":"Lerche","year":"2021","journal-title":"Mov. Disord."},{"key":"10.1016\/j.ymthe.2025.12.011_bib20","doi-asserted-by":"crossref","first-page":"416","DOI":"10.1002\/mds.28846","article-title":"Plasma Glucosylsphingosine in GBA1 Mutation Carriers with and without Parkinson's Disease","volume":"37","author":"Surface","year":"2022","journal-title":"Mov. Disord."},{"key":"10.1016\/j.ymthe.2025.12.011_bib21","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1038\/s41531-022-00363-2","article-title":"Elevation of gangliosides in four brain regions from Parkinson's disease patients with a GBA mutation","volume":"8","author":"Blumenreich","year":"2022","journal-title":"NPJ Parkinsons Dis."},{"key":"10.1016\/j.ymthe.2025.12.011_bib22","doi-asserted-by":"crossref","first-page":"12101","DOI":"10.1073\/pnas.1108197108","article-title":"CNS expression of glucocerebrosidase corrects alpha-synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathy","volume":"108","author":"Sardi","year":"2011","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2025.12.011_bib23","doi-asserted-by":"crossref","first-page":"3537","DOI":"10.1073\/pnas.1220464110","article-title":"Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathies","volume":"110","author":"Sardi","year":"2013","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2025.12.011_bib24","doi-asserted-by":"crossref","first-page":"S183","DOI":"10.3233\/JPD-212739","article-title":"Gene Therapy for Parkinson's Disease Associated with GBA1 Mutations","volume":"11","author":"Abeliovich","year":"2021","journal-title":"J. Parkinsons Dis."},{"key":"10.1016\/j.ymthe.2025.12.011_bib25","article-title":"AAV-mediated ARSA replacement for the treatment of Metachromatic Leukodystrophy","author":"Ramachandran","year":"2025","journal-title":"bioRxiv"},{"key":"10.1016\/j.ymthe.2025.12.011_bib26","doi-asserted-by":"crossref","DOI":"10.1038\/npjparkd.2016.25","article-title":"Parkinson's disease as a system-level disorder","volume":"2","author":"Caligiore","year":"2016","journal-title":"NPJ Parkinsons Dis."},{"key":"10.1016\/j.ymthe.2025.12.011_bib27","doi-asserted-by":"crossref","first-page":"200","DOI":"10.1007\/s10930-019-09838-3","article-title":"A Brief History of Protein Sorting Prediction","volume":"38","author":"Nielsen","year":"2019","journal-title":"Protein J."},{"key":"10.1016\/j.ymthe.2025.12.011_bib28","doi-asserted-by":"crossref","first-page":"907","DOI":"10.1038\/nchembio.466","article-title":"Ultrasensitive in situ visualization of active glucocerebrosidase molecules","volume":"6","author":"Witte","year":"2010","journal-title":"Nat. Chem. Biol."},{"key":"10.1016\/j.ymthe.2025.12.011_bib29","doi-asserted-by":"crossref","first-page":"550","DOI":"10.1089\/ars.2015.6307","article-title":"Sustained Systemic Glucocerebrosidase Inhibition Induces Brain alpha-Synuclein Aggregation, Microglia and Complement C1q Activation in Mice","volume":"23","author":"Rocha","year":"2015","journal-title":"Antioxid. Redox Signal."},{"key":"10.1016\/j.ymthe.2025.12.011_bib30","doi-asserted-by":"crossref","first-page":"2093","DOI":"10.1016\/S0002-9440(10)63566-3","article-title":"Viable mouse models of acid beta-glucosidase deficiency: the defect in Gaucher disease","volume":"163","author":"Xu","year":"2003","journal-title":"Am. J. Pathol."},{"key":"10.1016\/j.ymthe.2025.12.011_bib31","doi-asserted-by":"crossref","DOI":"10.1016\/j.neuint.2019.104502","article-title":"Age-related neurochemical and behavioural changes in D409V\/WT GBA1 mouse: Relevance to lewy body dementia","volume":"129","author":"Clarke","year":"2019","journal-title":"Neurochem. Int."},{"key":"10.1016\/j.ymthe.2025.12.011_bib32","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0252325","article-title":"Decreased glucocerebrosidase activity and substrate accumulation of glycosphingolipids in a novel GBA1 D409V knock-in mouse model","volume":"16","author":"Polinski","year":"2021","journal-title":"PLoS One"},{"key":"10.1016\/j.ymthe.2025.12.011_bib33","doi-asserted-by":"crossref","first-page":"843","DOI":"10.1093\/hmg\/ddt468","article-title":"Neuronal accumulation of glucosylceramide in a mouse model of neuronopathic Gaucher disease leads to neurodegeneration","volume":"23","author":"Farfel-Becker","year":"2014","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2025.12.011_bib34","doi-asserted-by":"crossref","first-page":"716","DOI":"10.1002\/1873-3468.12104","article-title":"Lysosomal glycosphingolipid catabolism by acid ceramidase: formation of glycosphingoid bases during deficiency of glycosidases","volume":"590","author":"Ferraz","year":"2016","journal-title":"FEBS Lett."},{"key":"10.1016\/j.ymthe.2025.12.011_bib35","doi-asserted-by":"crossref","first-page":"2595","DOI":"10.1002\/mds.28704","article-title":"CSF Protein Level of Neurotransmitter Secretion, Synaptic Plasticity, and Autophagy in PD and DLB","volume":"36","author":"Lerche","year":"2021","journal-title":"Mov. Disord."},{"key":"10.1016\/j.ymthe.2025.12.011_bib36","doi-asserted-by":"crossref","first-page":"74","DOI":"10.1038\/s41531-023-00517-w","article-title":"Glucocerebrosidase activity and lipid levels are related to protein pathologies in Parkinson's disease","volume":"9","author":"Leyns","year":"2023","journal-title":"NPJ Parkinsons Dis."},{"key":"10.1016\/j.ymthe.2025.12.011_bib37","doi-asserted-by":"crossref","first-page":"33","DOI":"10.1038\/s41531-024-00645-x","article-title":"Large-scale proteomics analysis of five brain regions from Parkinson's disease patients with a GBA1 mutation","volume":"10","author":"Blumenreich","year":"2024","journal-title":"NPJ Parkinsons Dis."},{"key":"10.1016\/j.ymthe.2025.12.011_bib38","doi-asserted-by":"crossref","first-page":"A2","DOI":"10.1259\/bjr.75.suppl_1.750002","article-title":"Skeletal aspects of Gaucher disease: a review","volume":"75","author":"Wenstrup","year":"2002","journal-title":"Br. J. Radiol."},{"key":"10.1016\/j.ymthe.2025.12.011_bib39","doi-asserted-by":"crossref","first-page":"438","DOI":"10.1016\/j.ymgme.2011.08.004","article-title":"Therapeutic approaches to bone pathology in Gaucher disease: past, present and future","volume":"104","author":"Goker-Alpan","year":"2011","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2025.12.011_bib40","doi-asserted-by":"crossref","first-page":"996","DOI":"10.1002\/jbmr.3734","article-title":"Gaucher Disease in Bone: From Pathophysiology to Practice","volume":"34","author":"Hughes","year":"2019","journal-title":"J. Bone Miner. Res."},{"key":"10.1016\/j.ymthe.2025.12.011_bib41","doi-asserted-by":"crossref","first-page":"2255","DOI":"10.1093\/brain\/awy147","article-title":"Is Parkinson's disease a lysosomal disorder?","volume":"141","author":"Klein","year":"2018","journal-title":"Brain"},{"key":"10.1016\/j.ymthe.2025.12.011_bib42","doi-asserted-by":"crossref","first-page":"1304","DOI":"10.1093\/brain\/awu002","article-title":"Glucocerebrosidase is shaking up the synucleinopathies","volume":"137","author":"Siebert","year":"2014","journal-title":"Brain"},{"key":"10.1016\/j.ymthe.2025.12.011_bib43","doi-asserted-by":"crossref","first-page":"31918","DOI":"10.1074\/jbc.M302752200","article-title":"Saposin C is required for normal resistance of acid beta-glucosidase to proteolytic degradation","volume":"278","author":"Sun","year":"2003","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.ymthe.2025.12.011_bib44","doi-asserted-by":"crossref","first-page":"5571","DOI":"10.1038\/s41598-019-41914-7","article-title":"Combination of acid beta-glucosidase mutation and Saposin C deficiency in mice reveals Gba1 mutation dependent and tissue-specific disease phenotype","volume":"9","author":"Liou","year":"2019","journal-title":"Sci. Rep."},{"key":"10.1016\/j.ymthe.2025.12.011_bib45","doi-asserted-by":"crossref","first-page":"168","DOI":"10.1002\/ana.20338","article-title":"Microglial activation and dopamine terminal loss in early Parkinson's disease","volume":"57","author":"Ouchi","year":"2005","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.ymthe.2025.12.011_bib46","doi-asserted-by":"crossref","first-page":"404","DOI":"10.1016\/j.nbd.2005.08.002","article-title":"In vivo imaging of microglial activation with [11C](R)-PK11195 PET in idiopathic Parkinson's disease","volume":"21","author":"Gerhard","year":"2006","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.ymthe.2025.12.011_bib47","doi-asserted-by":"crossref","first-page":"382","DOI":"10.1016\/S1474-4422(09)70062-6","article-title":"Neuroinflammation in Parkinson's disease: a target for neuroprotection?","volume":"8","author":"Hirsch","year":"2009","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.ymthe.2025.12.011_bib48","doi-asserted-by":"crossref","first-page":"579","DOI":"10.1007\/s12149-016-1099-2","article-title":"Extrastriatal spreading of microglial activation in Parkinson's disease: a positron emission tomography study","volume":"30","author":"Terada","year":"2016","journal-title":"Ann. Nucl. Med."},{"key":"10.1016\/j.ymthe.2025.12.011_bib49","doi-asserted-by":"crossref","DOI":"10.1016\/j.nbd.2019.104647","article-title":"A characterization of Gaucher iPS-derived astrocytes: Potential implications for Parkinson's disease","volume":"134","author":"Aflaki","year":"2020","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.ymthe.2025.12.011_bib50","doi-asserted-by":"crossref","first-page":"941","DOI":"10.1016\/j.cmet.2013.04.014","article-title":"Mitochondria and quality control defects in a mouse model of Gaucher disease--links to Parkinson's disease","volume":"17","author":"Osellame","year":"2013","journal-title":"Cell Metab."},{"key":"10.1016\/j.ymthe.2025.12.011_bib51","doi-asserted-by":"crossref","first-page":"220","DOI":"10.1186\/s12974-021-02272-2","article-title":"Inhibition of microglial beta-glucocerebrosidase hampers the microglia-mediated antioxidant and protective response in neurons","volume":"18","author":"Brunialti","year":"2021","journal-title":"J. Neuroinflammation"},{"key":"10.1016\/j.ymthe.2025.12.011_bib52","doi-asserted-by":"crossref","DOI":"10.7554\/eLife.79830","article-title":"Neuroinflammation in neuronopathic Gaucher disease: Role of microglia and NK cells, biomarkers, and response to substrate reduction therapy","volume":"11","author":"Boddupalli","year":"2022","journal-title":"Elife"},{"key":"10.1016\/j.ymthe.2025.12.011_bib53","doi-asserted-by":"crossref","first-page":"774","DOI":"10.1002\/mds.28375","article-title":"Brain Microglial Activation Increased in Glucocerebrosidase (GBA) Mutation Carriers without Parkinson's disease","volume":"36","author":"Mullin","year":"2021","journal-title":"Mov. Disord."},{"key":"10.1016\/j.ymthe.2025.12.011_bib54","doi-asserted-by":"crossref","first-page":"1023","DOI":"10.1038\/s41587-021-01156-3","article-title":"SignalP 6.0 predicts all five types of signal peptides using protein language models","volume":"40","author":"Teufel","year":"2022","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2025.12.011_bib55","doi-asserted-by":"crossref","first-page":"3387","DOI":"10.1093\/bioinformatics\/btx431","article-title":"DeepLoc: prediction of protein subcellular localization using deep learning","volume":"33","author":"Almagro Armenteros","year":"2017","journal-title":"Bioinformatics"},{"key":"10.1016\/j.ymthe.2025.12.011_bib56","doi-asserted-by":"crossref","first-page":"33","DOI":"10.1016\/j.omtm.2017.12.004","article-title":"Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes","volume":"9","author":"Nass","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2025.12.011_bib57","article-title":"Purification of Recombinant Adeno-Associated Viruses (rAAVs) by Cesium Chloride Gradient Sedimentation","volume":"2020","author":"Su","year":"2020","journal-title":"Cold Spring Harb. Protoc."}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001625010342?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001625010342?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,4,16]],"date-time":"2026-04-16T20:36:35Z","timestamp":1776371795000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001625010342"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,3]]},"references-count":57,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2026,3]]}},"alternative-id":["S1525001625010342"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2025.12.011","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2026,3]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"AAV gene therapy for GBA1-related diseases","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2025.12.011","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 Sanofi. Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}]}}