{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T14:21:22Z","timestamp":1790778082357,"version":"4.1.0"},"reference-count":67,"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":[[2021,10,5]],"date-time":"2021-10-05T00:00:00Z","timestamp":1633392000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100012479","name":"Weston Brain Institute","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100012479","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000024","name":"Canadian Institutes of Health Research","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000024","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000143","name":"Alzheimer Society of Canada","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000143","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100002808","name":"Carlsbergfondet","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100002808","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000070","name":"National Institute of Biomedical Imaging and Bioengineering","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000070","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.2021.10.001","type":"journal-article","created":{"date-parts":[[2021,10,9]],"date-time":"2021-10-09T12:23:12Z","timestamp":1633782192000},"page":"390-405","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":31,"special_numbering":"C","title":["Transgene distribution and immune response after ultrasound delivery of rAAV9 and PHP.B to the brain in a mouse model of amyloidosis"],"prefix":"10.1016","volume":"23","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0177-0739","authenticated-orcid":false,"given":"Rikke Hahn","family":"Kofoed","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stefan","family":"Heinen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4364-5277","authenticated-orcid":false,"given":"Joseph","family":"Silburt","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sonam","family":"Dubey","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7306-8087","authenticated-orcid":false,"given":"Chinaza Lilian","family":"Dibia","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miriam","family":"Maes","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0654-4303","authenticated-orcid":false,"given":"Elizabeth M.","family":"Simpson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kullervo","family":"Hynynen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5163-9961","authenticated-orcid":false,"given":"Isabelle","family":"Aubert","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2021.10.001_bib1","doi-asserted-by":"crossref","first-page":"834","DOI":"10.1001\/jamaneurol.2018.0233","article-title":"Adeno-Associated Viral Vector (Serotype 2)-Nerve Growth Factor for Patients With Alzheimer Disease: A Randomized Clinical Trial","volume":"75","author":"Rafii","year":"2018","journal-title":"JAMA Neurol."},{"key":"10.1016\/j.omtm.2021.10.001_bib2","doi-asserted-by":"crossref","first-page":"1139","DOI":"10.1001\/jamaneurol.2015.1807","article-title":"Nerve Growth Factor Gene Therapy: Activation of Neuronal Responses in Alzheimer Disease","volume":"72","author":"Tuszynski","year":"2015","journal-title":"JAMA Neurol."},{"key":"10.1016\/j.omtm.2021.10.001_bib3","doi-asserted-by":"crossref","first-page":"551","DOI":"10.1038\/nm1239","article-title":"A phase 1 clinical trial of nerve growth factor gene therapy for Alzheimer disease","volume":"11","author":"Tuszynski","year":"2005","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2021.10.001_bib4","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1089\/hum.2019.367","article-title":"Postmortem analysis in a clinical trial of AAV2-NGF gene therapy for alzheimer\u2019s disease identifies a need for improved vector delivery","volume":"31","author":"Castle","year":"2020","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2021.10.001_bib5","doi-asserted-by":"crossref","first-page":"24","DOI":"10.1089\/humc.2017.231","article-title":"AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer\u2019s Disease","volume":"29","author":"Rosenberg","year":"2018","journal-title":"Hum. Gene Ther. Clin. Dev."},{"key":"10.1016\/j.omtm.2021.10.001_bib6","doi-asserted-by":"crossref","first-page":"1058","DOI":"10.1038\/mt.2011.72","article-title":"Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates","volume":"19","author":"Gray","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.10.001_bib7","doi-asserted-by":"crossref","first-page":"328","DOI":"10.1089\/hum.2016.172","article-title":"Impact of AAV Capsid-Specific T-Cell Responses on Design and Outcome of Clinical Gene Transfer Trials with Recombinant Adeno-Associated Viral Vectors: An Evolving Controversy","volume":"28","author":"Ertl","year":"2017","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2021.10.001_bib8","doi-asserted-by":"crossref","first-page":"1144","DOI":"10.1089\/hum.2012.013","article-title":"Targeted delivery of self-complementary adeno-associated virus serotype 9 to the brain, using magnetic resonance imaging-guided focused ultrasound","volume":"23","author":"Th\u00e9venot","year":"2012","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2021.10.001_bib9","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.omtm.2021.10.001_bib10","doi-asserted-by":"crossref","first-page":"640","DOI":"10.1148\/radiol.2202001804","article-title":"Noninvasive MR imaging-guided focal opening of the blood-brain barrier in rabbits","volume":"220","author":"Hynynen","year":"2001","journal-title":"Radiology"},{"key":"10.1016\/j.omtm.2021.10.001_bib11","doi-asserted-by":"crossref","first-page":"2336","DOI":"10.1038\/s41467-018-04529-6","article-title":"Blood-brain barrier opening in Alzheimer\u2019s disease using MR-guided focused ultrasound","volume":"9","author":"Lipsman","year":"2018","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2021.10.001_bib12","doi-asserted-by":"crossref","first-page":"e57682","DOI":"10.1371\/journal.pone.0057682","article-title":"Noninvasive and targeted gene delivery into the brain using microbubble-facilitated focused ultrasound","volume":"8","author":"Hsu","year":"2013","journal-title":"PLoS ONE"},{"key":"10.1016\/j.omtm.2021.10.001_bib13","doi-asserted-by":"crossref","first-page":"568","DOI":"10.1038\/gt.2015.25","article-title":"Gene delivery to the spinal cord using MRI-guided focused ultrasound","volume":"22","author":"Weber-Adrian","year":"2015","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2021.10.001_bib14","doi-asserted-by":"crossref","first-page":"104","DOI":"10.1038\/gt.2014.91","article-title":"Noninvasive, neuron-specific gene therapy can be facilitated by focused ultrasound and recombinant adeno-associated virus","volume":"22","author":"Wang","year":"2015","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2021.10.001_bib15","doi-asserted-by":"crossref","first-page":"8127","DOI":"10.7150\/thno.36718","article-title":"Strategy to enhance transgene expression in proximity of amyloid plaques in a mouse model of Alzheimer\u2019s disease","volume":"9","author":"Weber-Adrian","year":"2019","journal-title":"Theranostics"},{"key":"10.1016\/j.omtm.2021.10.001_bib16","doi-asserted-by":"crossref","first-page":"912","DOI":"10.1016\/j.ymthe.2019.02.013","article-title":"The GPI-Linked Protein LY6A Drives AAV-PHP.B Transport across the Blood-Brain Barrier","volume":"27","author":"Hordeaux","year":"2019","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.10.001_bib17","doi-asserted-by":"crossref","first-page":"e0225206","DOI":"10.1371\/journal.pone.0225206","article-title":"Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids","volume":"14","author":"Huang","year":"2019","journal-title":"PLoS ONE"},{"key":"10.1016\/j.omtm.2021.10.001_bib18","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."},{"key":"10.1016\/j.omtm.2021.10.001_bib19","doi-asserted-by":"crossref","first-page":"182","DOI":"10.1016\/j.neulet.2017.11.049","article-title":"Intravenous administration of the adeno-associated virus-PHP.B capsid fails to upregulate transduction efficiency in the marmoset brain","volume":"665","author":"Matsuzaki","year":"2018","journal-title":"Neurosci. Lett."},{"key":"10.1016\/j.omtm.2021.10.001_bib20","doi-asserted-by":"crossref","first-page":"664","DOI":"10.1016\/j.ymthe.2018.01.018","article-title":"The neurotropic properties of AAV-PHP.B are limited to C57BL\/6J mice","volume":"26","author":"Hordeaux","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.10.001_bib21","doi-asserted-by":"crossref","first-page":"2697","DOI":"10.1093\/brain\/awt188","article-title":"Contribution of cerebrovascular disease in autopsy confirmed neurodegenerative disease cases in the National Alzheimer\u2019s Coordinating Centre","volume":"136","author":"Toledo","year":"2013","journal-title":"Brain"},{"key":"10.1016\/j.omtm.2021.10.001_bib22","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.bbi.2016.07.003","article-title":"Inflammation in Alzheimer\u2019s disease: Lessons learned from microglia-depletion models","volume":"61","author":"Spangenberg","year":"2017","journal-title":"Brain Behav. Immun."},{"key":"10.1016\/j.omtm.2021.10.001_bib23","doi-asserted-by":"crossref","first-page":"21562","DOI":"10.1074\/jbc.M100710200","article-title":"Early-onset amyloid deposition and cognitive deficits in transgenic mice expressing a double mutant form of amyloid precursor protein 695","volume":"276","author":"Chishti","year":"2001","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2021.10.001_bib24","doi-asserted-by":"crossref","first-page":"595","DOI":"10.15252\/emmm.201606210","article-title":"The amyloid hypothesis of Alzheimer\u2019s disease at 25 years","volume":"8","author":"Selkoe","year":"2016","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.omtm.2021.10.001_bib25","doi-asserted-by":"crossref","first-page":"52","DOI":"10.1186\/s13041-016-0232-4","article-title":"rAAV-compatible MiniPromoters for restricted expression in the brain and eye","volume":"9","author":"de Leeuw","year":"2016","journal-title":"Mol. Brain"},{"key":"10.1016\/j.omtm.2021.10.001_bib26","doi-asserted-by":"crossref","first-page":"32691","DOI":"10.1073\/pnas.1908658117","article-title":"Clinically approved IVIg delivered to the hippocampus with focused ultrasound promotes neurogenesis in a model of Alzheimer\u2019s disease","volume":"117","author":"Dubey","year":"2020","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2021.10.001_bib27","doi-asserted-by":"crossref","first-page":"eaax6646","DOI":"10.1126\/sciadv.aax6646","article-title":"Focused ultrasound delivery of a selective TrkA agonist rescues cholinergic function in a mouse model of Alzheimer\u2019s disease","volume":"6","author":"Xhima","year":"2020","journal-title":"Sci. Adv."},{"key":"10.1016\/j.omtm.2021.10.001_bib28","doi-asserted-by":"crossref","first-page":"329","DOI":"10.1038\/mt.2013.266","article-title":"AAV9-mediated expression of a non-self protein in nonhuman primate central nervous system triggers widespread neuroinflammation driven by antigen-presenting cell transduction","volume":"22","author":"Samaranch","year":"2014","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.10.001_bib29","doi-asserted-by":"crossref","first-page":"158","DOI":"10.1038\/mt.2012.167","article-title":"Cerebral infusion of AAV9 vector-encoding non-self proteins can elicit cell-mediated immune responses","volume":"21","author":"Ciesielska","year":"2013","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.10.001_bib30","doi-asserted-by":"crossref","first-page":"225","DOI":"10.1089\/hum.2008.151","article-title":"Transduction of nonhuman primate brain with adeno-associated virus serotype 1: vector trafficking and immune response","volume":"20","author":"Hadaczek","year":"2009","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2021.10.001_bib31","doi-asserted-by":"crossref","first-page":"465","DOI":"10.1016\/j.ymthe.2018.11.013","article-title":"AAV-Mediated Progranulin Delivery to a Mouse Model of Progranulin Deficiency Causes T Cell-Mediated Toxicity","volume":"27","author":"Amado","year":"2019","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.10.001_bib32","doi-asserted-by":"crossref","first-page":"2727","DOI":"10.1128\/JVI.01990-07","article-title":"Complement is an essential component of the immune response to adeno-associated virus vectors","volume":"82","author":"Zaiss","year":"2008","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.omtm.2021.10.001_bib33","doi-asserted-by":"crossref","first-page":"1061","DOI":"10.1089\/hum.2017.150","article-title":"Unraveling the complex story of immune responses to AAV vectors trial after trial","volume":"28","author":"Vandamme","year":"2017","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2021.10.001_bib34","article-title":"Not peer review manuscript: MORPHIOUS: A Machine Learning Workflow to Naively Detect the Activation of Microglia and Astrocytes","author":"Silburt","year":"2020","journal-title":"bioRxiv"},{"key":"10.1016\/j.omtm.2021.10.001_bib35","doi-asserted-by":"crossref","first-page":"197","DOI":"10.1385\/IR:36:1:197","article-title":"Complement-mediated activation of the adaptive immune responses: role of C3d in linking the innate and adaptive immunity","volume":"36","author":"Toapanta","year":"2006","journal-title":"Immunol. Res."},{"key":"10.1016\/j.omtm.2021.10.001_bib36","doi-asserted-by":"crossref","first-page":"10","DOI":"10.1186\/s40246-016-0074-2","article-title":"Organization, evolution and functions of the human and mouse Ly6\/uPAR family genes","volume":"10","author":"Loughner","year":"2016","journal-title":"Hum. Genomics"},{"key":"10.1016\/j.omtm.2021.10.001_bib37","doi-asserted-by":"crossref","first-page":"463","DOI":"10.1078\/0344-0338-00446","article-title":"Neuronal nuclear antigen (NeuN): a new tool in the diagnosis of central neurocytoma","volume":"199","author":"Soylemezoglu","year":"2003","journal-title":"Pathol. Res. Pract."},{"key":"10.1016\/j.omtm.2021.10.001_bib38","doi-asserted-by":"crossref","first-page":"1637","DOI":"10.1007\/s12035-015-9122-5","article-title":"Novel Insights into NeuN: from Neuronal Marker to Splicing Regulator","volume":"53","author":"Duan","year":"2016","journal-title":"Mol. Neurobiol."},{"key":"10.1016\/j.omtm.2021.10.001_bib39","doi-asserted-by":"crossref","first-page":"2","DOI":"10.1186\/1471-2202-8-2","article-title":"Evidence for a wide extra-astrocytic distribution of S100B in human brain","volume":"8","author":"Steiner","year":"2007","journal-title":"BMC Neurosci."},{"key":"10.1016\/j.omtm.2021.10.001_bib40","first-page":"9605265","article-title":"The Appropriate Marker for Astrocytes: Comparing the Distribution and Expression of Three Astrocytic Markers in Different Mouse Cerebral Regions","volume":"2019","author":"Zhang","year":"2019","journal-title":"BioMed Res. Int."},{"key":"10.1016\/j.omtm.2021.10.001_bib41","first-page":"1","article-title":"Blood-brain barrier disruption for the delivery of non-infectious viral vectors and proteins, preliminary study","author":"Foiret","year":"2017","journal-title":"IEEE Int. Ultrason. Symp. IUS"},{"key":"10.1016\/j.omtm.2021.10.001_bib42","doi-asserted-by":"crossref","first-page":"3631","DOI":"10.1038\/s41598-019-40071-1","article-title":"Vector uncoating limits adeno-associated viral vector-mediated transduction of human dendritic cells and vector immunogenicity","volume":"9","author":"Rossi","year":"2019","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtm.2021.10.001_bib43","doi-asserted-by":"crossref","first-page":"723","DOI":"10.1016\/j.ymthe.2019.12.010","article-title":"AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer","volume":"28","author":"Verdera","year":"2020","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.10.001_bib44","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.2021.10.001_bib45","doi-asserted-by":"crossref","first-page":"233","DOI":"10.1016\/j.omtm.2019.01.006","article-title":"Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid \u03b1-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease","volume":"12","author":"Lim","year":"2019","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2021.10.001_bib46","doi-asserted-by":"crossref","first-page":"16","DOI":"10.1016\/j.expneurol.2013.05.008","article-title":"Amyloid-\u03b2 plaque reduction, endogenous antibody delivery and glial activation by brain-targeted, transcranial focused ultrasound","volume":"248","author":"Jord\u00e3o","year":"2013","journal-title":"Exp. Neurol."},{"key":"10.1016\/j.omtm.2021.10.001_bib47","doi-asserted-by":"crossref","first-page":"930","DOI":"10.1089\/hum.2009.060","article-title":"Adeno-associated virus-mediated gene transfer to nonhuman primate liver can elicit destructive transgene-specific T\u00a0cell responses","volume":"20","author":"Gao","year":"2009","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2021.10.001_bib48","doi-asserted-by":"crossref","first-page":"612","DOI":"10.1038\/mt.2015.35","article-title":"Transduction of antigen-presenting cells in the brain by AAV9 warrants caution in preclinical studies","volume":"23","author":"Forsayeth","year":"2015","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.10.001_bib49","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.omtm.2021.10.001_bib50","doi-asserted-by":"crossref","first-page":"554","DOI":"10.3389\/fimmu.2018.00554","article-title":"Gene therapy with regulatory T\u00a0cells: A beneficial alliance","volume":"9","author":"Biswas","year":"2018","journal-title":"Front. Immunol."},{"key":"10.1016\/j.omtm.2021.10.001_bib51","doi-asserted-by":"crossref","first-page":"85","DOI":"10.1016\/j.omtm.2018.11.002","article-title":"AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice","volume":"12","author":"Colella","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2021.10.001_bib52","doi-asserted-by":"crossref","first-page":"434","DOI":"10.1016\/j.omtm.2021.03.021","article-title":"Synapsin-caveolin-1 gene therapy preserves neuronal and synaptic morphology and prevents neurodegeneration in a mouse model of AD","volume":"21","author":"Wang","year":"2021","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2021.10.001_bib53","first-page":"116","article-title":"Better Targeting, Better Efficiency for Wide-Scale Neuronal Transduction with the Synapsin Promoter and AAV-PHP.B","volume":"9","author":"Jackson","year":"2016","journal-title":"Front. Mol. Neurosci."},{"key":"10.1016\/j.omtm.2021.10.001_bib54","doi-asserted-by":"crossref","first-page":"599","DOI":"10.1089\/hum.2020.301","article-title":"Intracerebroventricular Administration of AAV9-PHP.B SYN1-EmGFP Induces Widespread Transgene Expression in the Mouse and Monkey CNS","volume":"32","author":"Galvan","year":"2021","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2021.10.001_bib55","doi-asserted-by":"crossref","first-page":"1934","DOI":"10.1038\/s41598-021-81046-5","article-title":"Systemic AAV6-synapsin-GFP administration results in lower liver biodistribution, compared to AAV1&2 and AAV9, with neuronal expression following ultrasound-mediated brain delivery","volume":"11","author":"Weber-Adrian","year":"2021","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtm.2021.10.001_bib56","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1089\/hum.2018.118","article-title":"New MiniPromoter Ple345 (NEFL) Drives Strong and Specific Expression in Retinal Ganglion Cells of Mouse and Primate Retina","volume":"30","author":"Simpson","year":"2019","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2021.10.001_bib57","doi-asserted-by":"crossref","first-page":"351","DOI":"10.1038\/s41434-021-00227-z","article-title":"Human MiniPromoters for ocular-rAAV expression in ON bipolar, cone, corneal, endothelial, M\u00fcller glial, and PAX6 cells","volume":"28","author":"Korecki","year":"2021","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2021.10.001_bib58","doi-asserted-by":"crossref","first-page":"16051","DOI":"10.1038\/mtm.2016.51","article-title":"PAX6 MiniPromoters drive restricted expression from rAAV in the adult mouse retina","volume":"3","author":"Hickmott","year":"2016","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2021.10.001_bib59","doi-asserted-by":"crossref","first-page":"1360","DOI":"10.1016\/j.bbagen.2007.06.005","article-title":"Investigations of combinations of mutations in the jellyfish green fluorescent protein (GFP) that afford brighter fluorescence, and use of a version (VisGreen) in plant, bacterial, and animal cells","volume":"1770","author":"Teerawanichpan","year":"2007","journal-title":"Biochim. Biophys. Acta"},{"key":"10.1016\/j.omtm.2021.10.001_bib60","doi-asserted-by":"crossref","first-page":"605","DOI":"10.1038\/gt.2009.3","article-title":"Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS","volume":"16","author":"Zanta-Boussif","year":"2009","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2021.10.001_bib61","doi-asserted-by":"crossref","first-page":"2886","DOI":"10.1128\/JVI.73.4.2886-2892.1999","article-title":"Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors","volume":"73","author":"Zufferey","year":"1999","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.omtm.2021.10.001_bib62","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.omtm.2021.10.001_bib63","doi-asserted-by":"crossref","first-page":"177","DOI":"10.1007\/978-1-4939-9139-6_10","article-title":"MRI-guided focused ultrasound for targeted delivery of rAAV to the brain","volume":"1950","author":"Noroozian","year":"2019","journal-title":"Methods Mol. Biol."},{"key":"10.1016\/j.omtm.2021.10.001_bib64","doi-asserted-by":"crossref","first-page":"2982","DOI":"10.7150\/thno.42611","article-title":"Focused ultrasound as a novel strategy for noninvasive gene delivery to retinal M\u00fcller glia","volume":"10","author":"Touahri","year":"2020","journal-title":"Theranostics"},{"key":"10.1016\/j.omtm.2021.10.001_bib65","doi-asserted-by":"crossref","first-page":"96","DOI":"10.1148\/radiol.11111417","article-title":"Blood-brain barrier: real-time feedback-controlled focused ultrasound disruption by using an acoustic emissions-based controller","volume":"263","author":"O\u2019Reilly","year":"2012","journal-title":"Radiology"},{"key":"10.1016\/j.omtm.2021.10.001_bib66","doi-asserted-by":"crossref","first-page":"3532","DOI":"10.1093\/bioinformatics\/btw413","article-title":"MorphoLibJ: integrated library and plugins for mathematical morphology with ImageJ","volume":"32","author":"Legland","year":"2016","journal-title":"Bioinformatics"},{"key":"10.1016\/j.omtm.2021.10.001_bib67","doi-asserted-by":"crossref","first-page":"989","DOI":"10.3171\/2017.8.JNS17790","article-title":"Safe and stable noninvasive focal gene delivery to the mammalian brain following focused ultrasound","volume":"130","author":"Stavarache","year":"2018","journal-title":"J.\u00a0Neurosurg."}],"container-title":["Molecular Therapy - Methods &amp; Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050121001583?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050121001583?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,6]],"date-time":"2025-11-06T09:30:39Z","timestamp":1762421439000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050121001583"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,12]]},"references-count":67,"alternative-id":["S2329050121001583"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2021.10.001","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":"Transgene distribution and immune response after ultrasound delivery of rAAV9 and PHP.B to the brain in a mouse model of amyloidosis","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2021.10.001","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2021 The Author(s).","name":"copyright","label":"Copyright"}]}}