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Ther."},{"issue":"13","key":"10.1016\/j.jconrel.2023.01.067_bb0300","doi-asserted-by":"crossref","first-page":"3247","DOI":"10.1002\/j.1460-2075.1996.tb00689.x","article-title":"\u201cMolecular mechanism and species specificity of TAP inhibition by herpes simplex virus ICP47,\u201d (in eng)","volume":"15","author":"Ahn","year":"1996","journal-title":"EMBO J."},{"issue":"5","key":"10.1016\/j.jconrel.2023.01.067_bb0305","doi-asserted-by":"crossref","first-page":"613","DOI":"10.1016\/S1074-7613(00)80349-0","article-title":"\u201cThe ER-luminal domain of the HCMV glycoprotein US6 inhibits peptide translocation by TAP,\u201d (in eng)","volume":"6","author":"Ahn","year":"1997","journal-title":"Immunity"},{"issue":"13","key":"10.1016\/j.jconrel.2023.01.067_bb0310","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.99052","article-title":"\"Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity,\" (in eng)","volume":"5","author":"Xiao","year":"2019","journal-title":"JCI Insight"},{"issue":"569","key":"10.1016\/j.jconrel.2023.01.067_bb0315","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.aba9188","article-title":"MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates, (in eng)","volume":"12","author":"Hordeaux","year":"2020","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.jconrel.2023.01.067_bb0320","doi-asserted-by":"crossref","DOI":"10.1016\/j.biotechadv.2021.107764","article-title":"\"Cellular pathways of recombinant adeno-associated virus production for gene therapy,\" (in eng)","volume":"49","author":"Sha","year":"2021","journal-title":"Biotechnol. Adv."},{"key":"10.1016\/j.jconrel.2023.01.067_bb0325","doi-asserted-by":"crossref","DOI":"10.1016\/j.copbio.2022.102721","article-title":"\u201cGene therapy: challenges in cell culture scale-up,\u201d (in eng)","volume":"75","author":"Shupe","year":"2022","journal-title":"Curr. Opin. Biotechnol."},{"issue":"11","key":"10.1016\/j.jconrel.2023.01.067_bb0330","doi-asserted-by":"crossref","first-page":"1079","DOI":"10.1089\/104303403322124792","article-title":"\u201cPhase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study,\u201d (in eng)","volume":"14","author":"Flotte","year":"2003","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.jconrel.2023.01.067_bb0335","doi-asserted-by":"crossref","first-page":"166","DOI":"10.1016\/j.omtm.2018.01.002","article-title":"\"Pharmacology of recombinant adeno-associated virus production,\" (in eng)","volume":"8","author":"Penaud-Budloo","year":"2018","journal-title":"Mol. Ther. Meth. Clin. Dev."},{"issue":"11","key":"10.1016\/j.jconrel.2023.01.067_bb0340","doi-asserted-by":"crossref","first-page":"840","DOI":"10.1038\/gt.2008.65","article-title":"\u201cManufacturing and characterizing AAV-based vectors for use in clinical studies,\u201d (in eng)","volume":"15","author":"Wright","year":"2008","journal-title":"Gene Ther."},{"issue":"1","key":"10.1016\/j.jconrel.2023.01.067_bb0345","doi-asserted-by":"crossref","first-page":"80","DOI":"10.3390\/biomedicines2010080","article-title":"Product-related impurities in clinical-grade recombinant AAV vectors: characterization and risk assessment, (in eng)","volume":"2","author":"Wright","year":"2014","journal-title":"Biomedicines"},{"key":"10.1016\/j.jconrel.2023.01.067_bb0350","doi-asserted-by":"crossref","first-page":"271","DOI":"10.1016\/j.copbio.2021.12.009","article-title":"\u201cLeveraging rAAV bioprocess understanding and next generation bioanalytics development,\u201d (in eng)","volume":"74","author":"Escandell","year":"2022","journal-title":"Curr. Opin. Biotechnol."},{"issue":"7","key":"10.1016\/j.jconrel.2023.01.067_bb0355","doi-asserted-by":"crossref","first-page":"598","DOI":"10.1016\/j.addr.2011.10.005","article-title":"\u201cGuided delivery of adeno-associated viral vectors into the primate brain,\u201d (in eng)","volume":"64","author":"Salegio","year":"2012","journal-title":"Adv. Drug Deliv. Rev."},{"issue":"2","key":"10.1016\/j.jconrel.2023.01.067_bb0360","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1159\/000342492","article-title":"\u201cPathways of infusate loss during convection-enhanced delivery into the putamen nucleus,\u201d (in eng)","volume":"91","author":"Brady","year":"2013","journal-title":"Stereotact. Funct. Neurosurg."},{"issue":"11","key":"10.1016\/j.jconrel.2023.01.067_bb0365","doi-asserted-by":"crossref","first-page":"1210","DOI":"10.1136\/jnnp-2020-322904","article-title":"\u201cData-driven evolution of neurosurgical gene therapy delivery in Parkinson\u2019s disease,\u201d (in eng)","volume":"91","author":"Richardson","year":"2020","journal-title":"J. Neurol. Neurosurg. Psychiatry"},{"issue":"5","key":"10.1016\/j.jconrel.2023.01.067_bb0370","doi-asserted-by":"crossref","first-page":"463","DOI":"10.1089\/hum.2008.022","article-title":"\u201cTreatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA,\u201d (in eng)","volume":"19","author":"Worgall","year":"2008","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.jconrel.2023.01.067_bb0375","series-title":"Intracerebral Gene Therapy in Children with Metachromatic Leukodystrophy: Results of a Phase I\/II Trial","author":"Sevin","year":"2018"},{"issue":"17","key":"10.1016\/j.jconrel.2023.01.067_bb0380","doi-asserted-by":"crossref","DOI":"10.3390\/ijms22179200","article-title":"Gene therapy for neuronopathic mucopolysaccharidoses: state of the art, (in eng)","volume":"22","author":"de Castro","year":"2021","journal-title":"Int. J. Mol. Sci."},{"issue":"7","key":"10.1016\/j.jconrel.2023.01.067_bb0385","doi-asserted-by":"crossref","first-page":"478","DOI":"10.1089\/hum.2016.087","article-title":"\u201cAdeno-associated virus-based gene therapy for CNS diseases,\u201d (in eng)","volume":"27","author":"Hocquemiller","year":"2016","journal-title":"Hum. Gene Ther."},{"issue":"3","key":"10.1016\/j.jconrel.2023.01.067_bb0390","doi-asserted-by":"crossref","first-page":"348","DOI":"10.1038\/gt.2012.27","article-title":"\u201cAxonal transport of adeno-associated viral vectors is serotype-dependent,\u201d (in eng)","volume":"20","author":"Salegio","year":"2013","journal-title":"Gene Ther."},{"issue":"6","key":"10.1016\/j.jconrel.2023.01.067_bb0395","doi-asserted-by":"crossref","first-page":"520","DOI":"10.1038\/gt.2016.24","article-title":"\"Axonal transport of AAV9 in nonhuman primate brain,\" (in eng)","volume":"23","author":"Green","year":"2016","journal-title":"Gene Ther."},{"issue":"5","key":"10.1016\/j.jconrel.2023.01.067_bb0400","doi-asserted-by":"crossref","first-page":"922","DOI":"10.1038\/mt.2010.248","article-title":"\u201cAnterograde axonal transport of AAV2-GDNF in rat basal ganglia,\u201d (in eng)","volume":"19","author":"Ciesielska","year":"2011","journal-title":"Mol. Ther."},{"issue":"2","key":"10.1016\/j.jconrel.2023.01.067_bb0405","doi-asserted-by":"crossref","first-page":"228","DOI":"10.1016\/j.nbd.2011.10.004","article-title":"\u201cGlial-derived neurotrophic factor gene transfer for Parkinson\u2019s disease: anterograde distribution of AAV2 vectors in the primate brain,\u201d (in eng)","volume":"48","author":"Kells","year":"2012","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.jconrel.2023.01.067_bb0410","doi-asserted-by":"crossref","first-page":"434","DOI":"10.1016\/j.jconrel.2016.02.034","article-title":"AAV viral vector delivery to the brain by shape-conforming MR-guided infusions, (in eng)","volume":"240","author":"Bankiewicz","year":"2016","journal-title":"J. Controll. Releas.\u202f: Off. J. Controll. Release Soc."},{"issue":"4","key":"10.1016\/j.jconrel.2023.01.067_bb0415","doi-asserted-by":"crossref","first-page":"371","DOI":"10.1006\/mthe.2002.0564","article-title":"\u201cRecombinant adeno-associated virus serotypes 2- and 5-mediated gene transfer in the mammalian brain: quantitative analysis of heparin co-infusion,\u201d (in eng)","volume":"5","author":"Mastakov","year":"2002","journal-title":"Mol. Ther."},{"issue":"9579","key":"10.1016\/j.jconrel.2023.01.067_bb0420","first-page":"2097","article-title":"Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson\u2019s disease: an open label, phase I trial, (in eng)","volume":"369","author":"Kaplitt","year":"2007","journal-title":"s"},{"key":"10.1016\/j.jconrel.2023.01.067_bb0425","doi-asserted-by":"crossref","first-page":"71","DOI":"10.1016\/j.jconrel.2014.09.018","article-title":"Intra-arterial delivery of AAV vectors to the mouse brain after mannitol mediated blood brain barrier disruption, (in eng)","volume":"196","author":"Foley","year":"2014","journal-title":"J. Controll. Releas.\u202f: Off. J. Controll. Release Soc."},{"key":"10.1016\/j.jconrel.2023.01.067_bb0430","doi-asserted-by":"crossref","first-page":"217","DOI":"10.1007\/978-1-4939-3271-9_15","article-title":"\u201cMRI-Guided Delivery of Viral Vectors,\u201d (in eng)","volume":"1382","author":"Salegio","year":"2016","journal-title":"Meth. Mol. Biol. (Clifton, N.J.)"},{"issue":"4","key":"10.1016\/j.jconrel.2023.01.067_bb0435","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, (in eng)","volume":"20","author":"Gray","year":"2013","journal-title":"Gene Ther."},{"key":"10.1016\/j.jconrel.2023.01.067_bb0440","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1016\/j.omtm.2018.12.001","article-title":"\"Kinetics and MR-based monitoring of AAV9 vector delivery into cerebrospinal fluid of nonhuman primates,\" (in eng)","volume":"13","author":"Ohno","year":"2019","journal-title":"Mol. Ther. Meth. Clin. Dev."},{"issue":"2","key":"10.1016\/j.jconrel.2023.01.067_bb0445","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,\" (in eng)","volume":"28","author":"Taghian","year":"2020","journal-title":"Mol. Ther."},{"issue":"5","key":"10.1016\/j.jconrel.2023.01.067_bb0450","doi-asserted-by":"crossref","DOI":"10.1172\/JCI157820","article-title":"\u201cCLN7 gene therapy: hope for an ultra-rare condition,\u201d (in eng)","volume":"132","author":"Brudvig","year":"2022","journal-title":"J. Clin. Invest."},{"issue":"3","key":"10.1016\/j.jconrel.2023.01.067_bb0455","doi-asserted-by":"crossref","first-page":"477","DOI":"10.1038\/mt.2014.210","article-title":"\u201cImproving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates,\u201d (in eng)","volume":"23","author":"Meyer","year":"2015","journal-title":"Mol. Ther."},{"issue":"1","key":"10.1016\/j.jconrel.2023.01.067_bb0460","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,\" (in eng)","volume":"29","author":"Hinderer","year":"2018","journal-title":"Hum. Gene Ther."},{"issue":"7-8","key":"10.1016\/j.jconrel.2023.01.067_bb0465","doi-asserted-by":"crossref","first-page":"349","DOI":"10.1089\/hum.2020.105","article-title":"\"The challenge of gene therapy for neurological diseases: strategies and tools to achieve efficient delivery to the central nervous system,\" (in eng)","volume":"32","author":"Piguet","year":"2021","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.jconrel.2023.01.067_bb0470","doi-asserted-by":"crossref","first-page":"94","DOI":"10.1016\/j.jconrel.2016.09.011","article-title":"Gene therapy for the CNS using AAVs: The impact of systemic delivery by AAV9, (in eng)","volume":"241","author":"Saraiva","year":"2016","journal-title":"J. Controll. Releas.\u202f: Off. J. Controll. Release Soc."},{"issue":"7","key":"10.1016\/j.jconrel.2023.01.067_bb0475","doi-asserted-by":"crossref","first-page":"1299","DOI":"10.1038\/mt.2014.68","article-title":"\"Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10,\" (in eng)","volume":"22","author":"Yang","year":"2014","journal-title":"Mol. Ther."},{"issue":"1","key":"10.1016\/j.jconrel.2023.01.067_bb0480","doi-asserted-by":"crossref","first-page":"9844","DOI":"10.1038\/s41598-019-46069-z","article-title":"The adeno-associated virus rh10 vector is an effective gene transfer system for chronic spinal cord injury, (in eng)","volume":"9","author":"Hoshino","year":"2019","journal-title":"Sci. Rep."},{"key":"10.1016\/j.jconrel.2023.01.067_bb0485","doi-asserted-by":"crossref","DOI":"10.1016\/j.lfs.2021.119142","article-title":"\"Challenges in adeno-associated virus-based treatment of central nervous system diseases through systemic injection,\" (in eng)","volume":"270","author":"Huang","year":"2021","journal-title":"Life Sci."},{"issue":"6","key":"10.1016\/j.jconrel.2023.01.067_bb0490","doi-asserted-by":"crossref","first-page":"461","DOI":"10.1016\/j.tips.2021.03.004","article-title":"\u201cDelivering AAV to the Central Nervous and Sensory Systems,\u201d (in eng)","volume":"42","author":"Peters","year":"2021","journal-title":"Trends Pharmacol. Sci."},{"issue":"3","key":"10.1016\/j.jconrel.2023.01.067_bb0495","doi-asserted-by":"crossref","first-page":"640","DOI":"10.1148\/radiol.2202001804","article-title":"\u201cNoninvasive MR imaging-guided focal opening of the blood-brain barrier in rabbits,\u201d (in eng)","volume":"220","author":"Hynynen","year":"2001","journal-title":"Radiology"},{"issue":"7","key":"10.1016\/j.jconrel.2023.01.067_bb0500","doi-asserted-by":"crossref","first-page":"619","DOI":"10.1089\/hum.2014.011","article-title":"\u201cTranslational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy,\u201d (in eng)","volume":"25","author":"Passini","year":"2014","journal-title":"Hum. Gene Ther."},{"issue":"2","key":"10.1016\/j.jconrel.2023.01.067_bb0505","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.1500078","article-title":"\"Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model,\" (in eng)","volume":"1","author":"Nizzardo","year":"2015","journal-title":"Sci. Adv."},{"issue":"11","key":"10.1016\/j.jconrel.2023.01.067_bb0510","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,\" (in eng)","volume":"28","author":"Vandamme","year":"2017","journal-title":"Hum. Gene Ther."},{"issue":"2","key":"10.1016\/j.jconrel.2023.01.067_bb0515","first-page":"319","article-title":"\u201cSerologic evidence for human infection with adenovirus-associated viruses,\u201d (in eng)","volume":"40","author":"Blacklow","year":"1968","journal-title":"J. Natl. Cancer Inst."},{"issue":"3","key":"10.1016\/j.jconrel.2023.01.067_bb0520","doi-asserted-by":"crossref","first-page":"181","DOI":"10.2174\/156652311795684759","article-title":"\u201cAdeno-Associated Virus (AAV) Vectors in the CNS,\u201d (in eng)","volume":"11","author":"McCown","year":"2011","journal-title":"Current Gene Therap."},{"issue":"18","key":"10.1016\/j.jconrel.2023.01.067_bb0525","doi-asserted-by":"crossref","first-page":"11854","DOI":"10.1073\/pnas.182412299","article-title":"\u201cNovel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy,\u201d (in eng)","volume":"99","author":"Gao","year":"2002","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"issue":"1","key":"10.1016\/j.jconrel.2023.01.067_bb0530","doi-asserted-by":"crossref","first-page":"23","DOI":"10.1182\/blood-2013-01-306647","article-title":"\u201cImmune responses to AAV vectors: overcoming barriers to successful gene therapy,\u201d (in eng)","volume":"122","author":"Mingozzi","year":"2013","journal-title":"Blood"},{"issue":"2","key":"10.1016\/j.jconrel.2023.01.067_bb0535","doi-asserted-by":"crossref","first-page":"79","DOI":"10.1089\/humc.2016.048","article-title":"\u201cAAV natural infection induces broad cross-neutralizing antibody responses to multiple AAV serotypes in chimpanzees,\u201d (in eng)","volume":"27","author":"Calcedo","year":"2016","journal-title":"Human Gene Therapy. Clin. Develop."},{"key":"10.1016\/j.jconrel.2023.01.067_bb0540","doi-asserted-by":"crossref","first-page":"198","DOI":"10.1016\/j.omtm.2019.12.008","article-title":"\u201cImmune response mechanisms against AAV vectors in animal models,\u201d (in eng)","volume":"17","author":"Martino","year":"2020","journal-title":"Mol. Ther. Meth. Clin. Dev."},{"issue":"4","key":"10.1016\/j.jconrel.2023.01.067_bb0545","doi-asserted-by":"crossref","first-page":"413","DOI":"10.1002\/mus.27363","article-title":"\u201cExpert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy,\u201d (in eng)","volume":"64","author":"Kichula","year":"2021","journal-title":"Muscle Nerve"},{"key":"10.1016\/j.jconrel.2023.01.067_bb0550","article-title":"Management of neuroinflammatory responses to AAV-mediated gene therapies for neurodegenerative diseases, (in eng)","volume":"10, no. 2","author":"Perez","year":"2020","journal-title":"Brain Sci."},{"issue":"9","key":"10.1016\/j.jconrel.2023.01.067_bb0555","doi-asserted-by":"crossref","first-page":"1713","DOI":"10.1038\/mt.2012.114","article-title":"\u201cGene transfer to the CNS is efficacious in immune-primed mice harboring physiologically relevant titers of anti-AAV antibodies,\u201d (in eng)","volume":"20","author":"Treleaven","year":"2012","journal-title":"Mol. Ther."},{"issue":"2","key":"10.1016\/j.jconrel.2023.01.067_bb0560","doi-asserted-by":"crossref","first-page":"201","DOI":"10.1089\/10430349950018995","article-title":"\u201cAdeno-associated virus-mediated gene transfer to the brain: duration and modulation of expression,\u201d (in eng)","volume":"10","author":"Lo","year":"1999","journal-title":"Hum. Gene Ther."},{"issue":"11","key":"10.1016\/j.jconrel.2023.01.067_bb0565","doi-asserted-by":"crossref","first-page":"2084","DOI":"10.1038\/mt.2011.108","article-title":"\u201cA 10 patient case report on the impact of plasmapheresis upon neutralizing factors against adeno-associated virus (AAV) types 1, 2, 6, and 8,\u201d (in eng)","volume":"19","author":"Monteilhet","year":"2011","journal-title":"Mol. Ther."},{"issue":"194","key":"10.1016\/j.jconrel.2023.01.067_bb0570","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.3005795","article-title":"Overcoming preexisting humoral immunity to AAV using capsid decoys, (in eng)","volume":"5","author":"Mingozzi","year":"2013","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.jconrel.2023.01.067_bb0575","doi-asserted-by":"crossref","DOI":"10.3389\/fimmu.2021.655478","article-title":"\u201cCell-mediated immunity to NAGLU transgene following intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome,\u201d (in eng)","volume":"12","author":"Gougeon","year":"2021","journal-title":"Front. Immunol."},{"issue":"6","key":"10.1016\/j.jconrel.2023.01.067_bb0580","doi-asserted-by":"crossref","first-page":"845","DOI":"10.1517\/14712598.2015.1035645","article-title":"\u201cStrategies to circumvent humoral immunity to adeno-associated viral vectors,\u201d (in eng)","volume":"15","author":"Tse","year":"2015","journal-title":"Expert. Opin. Biol. Ther."},{"issue":"3","key":"10.1016\/j.jconrel.2023.01.067_bb0585","doi-asserted-by":"crossref","first-page":"723","DOI":"10.1016\/j.ymthe.2019.12.010","article-title":"\u201cAAV vector immunogenicity in humans: a long journey to successful gene transfer,\u201d (in eng)","volume":"28","author":"Verdera","year":"2020","journal-title":"Mol. Ther."},{"key":"10.1016\/j.jconrel.2023.01.067_bb0590","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1016\/j.copbio.2019.01.002","article-title":"\"Engineering the AAV capsid to evade immune responses,\" (in eng)","volume":"60","author":"Barnes","year":"2019","journal-title":"Curr. Opin. Biotechnol."},{"issue":"2","key":"10.1016\/j.jconrel.2023.01.067_bb0595","doi-asserted-by":"crossref","first-page":"379","DOI":"10.1016\/j.ymthe.2016.12.010","article-title":"\"Rescue of hearing by gene delivery to inner-ear hair cells using exosome-associated AAV,\" (in eng)","volume":"25","author":"Gy\u00f6rgy","year":"2017","journal-title":"Mol. 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Med."},{"issue":"11","key":"10.1016\/j.jconrel.2023.01.067_bb0710","doi-asserted-by":"crossref","first-page":"1391","DOI":"10.1089\/104303402760128612","article-title":"\u201cClinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain,\u201d (in eng)","volume":"13","author":"Janson","year":"2002","journal-title":"Hum. Gene Ther."},{"issue":"165","key":"10.1016\/j.jconrel.2023.01.067_bb0715","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.3003454","article-title":"Long-term follow-up after gene therapy for canavan disease, (in eng)","volume":"4","author":"Leone","year":"2012","journal-title":"Sci. Transl. Med."},{"issue":"1","key":"10.1016\/j.jconrel.2023.01.067_bb0720","doi-asserted-by":"crossref","first-page":"95","DOI":"10.1007\/s00401-017-1784-9","article-title":"\"Uncoupling N-acetylaspartate from brain pathology: implications for Canavan disease gene therapy,\" (in eng)","volume":"135","author":"von Jonquieres","year":"2018","journal-title":"Acta Neuropathol."},{"issue":"5","key":"10.1016\/j.jconrel.2023.01.067_bb0725","doi-asserted-by":"crossref","first-page":"277","DOI":"10.1038\/nrneurol.2013.56","article-title":"Progress in gene therapy for neurological disorders","volume":"9","author":"Simonato","year":"2013","journal-title":"Nat. Rev. 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Med."},{"issue":"6571","key":"10.1016\/j.jconrel.2023.01.067_bb0785","doi-asserted-by":"crossref","first-page":"252","DOI":"10.1038\/380252a0","article-title":"\u201cFunctional recovery in parkinsonian monkeys treated with GDNF,\u201d (in eng)","volume":"380","author":"Gash","year":"1996","journal-title":"Nature"},{"issue":"5","key":"10.1016\/j.jconrel.2023.01.067_bb0790","doi-asserted-by":"crossref","first-page":"589","DOI":"10.1038\/nm850","article-title":"\u201cDirect brain infusion of glial cell line-derived neurotrophic factor in Parkinson disease,\u201d (in eng)","volume":"9","author":"Gill","year":"2003","journal-title":"Nat. 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