{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,13]],"date-time":"2026-10-13T04:21:07Z","timestamp":1791865267389,"version":"4.3.4"},"reference-count":154,"publisher":"Springer Science and Business Media LLC","issue":"4","license":[{"start":{"date-parts":[[2025,7,15]],"date-time":"2025-07-15T00:00:00Z","timestamp":1752537600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"},{"start":{"date-parts":[[2025,7,15]],"date-time":"2025-07-15T00:00:00Z","timestamp":1752537600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"}],"funder":[{"DOI":"10.13039\/501100001809","name":"National Natural\u00a0Science Foundation of China","doi-asserted-by":"crossref","award":["81772833"],"award-info":[{"award-number":["81772833"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"crossref"}]},{"name":"Central Government Guides the Special Funds for the Development of Local Science and Technology","award":["2024BSB012"],"award-info":[{"award-number":["2024BSB012"]}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["CURR MED SCI"],"published-print":{"date-parts":[[2025,8]]},"DOI":"10.1007\/s11596-025-00084-6","type":"journal-article","created":{"date-parts":[[2025,7,15]],"date-time":"2025-07-15T15:29:34Z","timestamp":1752593374000},"page":"683-698","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Advances and Challenges in Adeno-Associated Virus Gene Therapy Applications of Localized Delivery Strategies"],"prefix":"10.1007","volume":"45","author":[{"ORCID":"https:\/\/orcid.org\/0009-0008-1477-2526","authenticated-orcid":false,"given":"Jin-hao","family":"Chen","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Li-jun","family":"Zhan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cun-ming","family":"Lv","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jun-bo","family":"Teng","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chun-yu","family":"Cao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,7,15]]},"reference":[{"issue":"3685","key":"84_CR1","doi-asserted-by":"publisher","first-page":"754","DOI":"10.1126\/science.149.3685.754","volume":"149","author":"RW Atchison","year":"1965","unstructured":"Atchison RW, Casto BC, Hammon WM. Adenovirus-associated defective virus particles. Science. 1965;149(3685):754\u2013756.","journal-title":"Science."},{"issue":"10","key":"84_CR2","first-page":"2072","volume":"4","author":"JD Tratschin","year":"1984","unstructured":"Tratschin JD, West MH, Sandbank T, et al. A human parvovirus, adeno-associated virus, as a eucaryotic vector: transient expression and encapsidation of the procaryotic gene for chloramphenicol acetyltransferase. Mol Cell Biol. 1984;4(10):2072\u20132081.","journal-title":"Mol Cell Biol."},{"issue":"20","key":"84_CR3","doi-asserted-by":"publisher","first-page":"6466","DOI":"10.1073\/pnas.81.20.6466","volume":"81","author":"PL Hermonat","year":"1984","unstructured":"Hermonat PL, Muzyczka N. Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells. Proc Natl Acad Sci USA. 1984;81(20):6466\u20136470.","journal-title":"Proc Natl Acad Sci USA"},{"issue":"9","key":"84_CR4","doi-asserted-by":"publisher","first-page":"1145","DOI":"10.1089\/hum.1996.7.9-1145","volume":"7","author":"T Flotte","year":"1996","unstructured":"Flotte T, Carter B, Conrad C, et al. A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease. Hum Gene Ther. 1996;7(9):1145\u20131159.","journal-title":"Hum Gene Ther."},{"issue":"4","key":"84_CR5","doi-asserted-by":"publisher","first-page":"347","DOI":"10.1007\/s40265-023-01845-0","volume":"83","author":"YA Heo","year":"2023","unstructured":"Heo YA. Etranacogene Dezaparvovec: First Approval. Drugs. 2023;83(4):347\u2013352.","journal-title":"Drugs."},{"issue":"14","key":"84_CR6","doi-asserted-by":"publisher","first-page":"1323","DOI":"10.1007\/s40265-023-01929-x","volume":"83","author":"SM Hoy","year":"2023","unstructured":"Hoy SM. Delandistrogene Moxeparvovec: First Approval. Drugs. 2023;83(14):1323\u20131329.","journal-title":"Drugs."},{"issue":"14","key":"84_CR7","doi-asserted-by":"publisher","first-page":"1505","DOI":"10.1007\/s40265-022-01788-y","volume":"82","author":"HA Blair","year":"2022","unstructured":"Blair HA. Valoctocogene Roxaparvovec: First Approval. Drugs. 2022;82(14):1505\u20131510.","journal-title":"Drugs."},{"issue":"1","key":"84_CR8","doi-asserted-by":"publisher","first-page":"601","DOI":"10.1146\/annurev-virology-092818-015530","volume":"6","author":"AM Keeler","year":"2019","unstructured":"Keeler AM, Flotte TR. Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here? Annu Rev Virol. 2019;6(1):601\u2013621.","journal-title":"Annu Rev Virol."},{"issue":"2","key":"84_CR9","doi-asserted-by":"publisher","first-page":"509","DOI":"10.1016\/j.ymthe.2021.11.005","volume":"30","author":"CH Tai","year":"2022","unstructured":"Tai CH, Lee NC, Chien YH, et al. Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency. Mol Ther. 2022;30(2):509\u2013518.","journal-title":"Mol Ther."},{"issue":"10097","key":"84_CR10","doi-asserted-by":"publisher","first-page":"849","DOI":"10.1016\/S0140-6736(17)31868-8","volume":"390","author":"S Russell","year":"2017","unstructured":"Russell S, Bennett J, Wellman JA, et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomized, controlled, open-label, phase 3 trial. Lancet. 2017;390(10097):849\u2013860.","journal-title":"Lancet."},{"issue":"9923","key":"84_CR11","doi-asserted-by":"publisher","first-page":"1129","DOI":"10.1016\/S0140-6736(13)62117-0","volume":"383","author":"RE MacLaren","year":"2014","unstructured":"MacLaren RE, Groppe M, Barnard AR, et al. Retinal gene therapy in patients with choroideremia: initial findings from a phase 1\/2 clinical trial. Lancet. 2014;383(9923):1129\u20131137.","journal-title":"Lancet."},{"issue":"11","key":"84_CR12","doi-asserted-by":"publisher","first-page":"923","DOI":"10.1182\/blood.2019003777","volume":"138","author":"F Leebeek","year":"2021","unstructured":"Leebeek F, Miesbach W. Gene therapy for hemophilia: a review on clinical benefit, limitations, and remaining issues. Blood. 2021;138(11):923\u2013931.","journal-title":"Blood."},{"issue":"10","key":"84_CR13","doi-asserted-by":"publisher","first-page":"2337","DOI":"10.1016\/j.ymthe.2018.07.011","volume":"26","author":"D Duan","year":"2018","unstructured":"Duan D. Systemic AAV Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy. Mol Ther. 2018;26(10):2337\u20132356.","journal-title":"Mol Ther."},{"issue":"17\u201318","key":"84_CR14","doi-asserted-by":"publisher","first-page":"927","DOI":"10.1089\/hum.2023.075","volume":"34","author":"N Mehta","year":"2023","unstructured":"Mehta N, Gilbert R, Chahal PS, et al. Preclinical Development and Characterization of Novel Adeno-Associated Viral Vectors for the Treatment of Lipoprotein Lipase Deficiency. Hum Gene Ther. 2023;34(17-18):927\u2013946.","journal-title":"Hum Gene Ther."},{"issue":"13\u201314","key":"84_CR15","doi-asserted-by":"publisher","first-page":"416","DOI":"10.1089\/hum.2023.162","volume":"35","author":"H Ertl","year":"2024","unstructured":"Ertl H. Circumventing B-Cell Responses to Allow for Redosing of Adeno-Associated Virus Vectors. Hum Gene Ther. 2024;35(13\u201314):416\u2013424.","journal-title":"Hum Gene Ther."},{"issue":"4","key":"84_CR16","doi-asserted-by":"publisher","first-page":"952","DOI":"10.1016\/j.ymthe.2024.02.002","volume":"32","author":"J Hordeaux","year":"2024","unstructured":"Hordeaux J, Lamontagne RJ, Song C, et al. High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury. Mol Ther. 2024;32(4):952\u2013968.","journal-title":"Mol Ther."},{"issue":"1","key":"84_CR17","doi-asserted-by":"publisher","first-page":"60","DOI":"10.1038\/s41434-023-00405-1","volume":"2","author":"D Stone","year":"2025","unstructured":"Stone D, Aubert M, Jerome KR. Adeno-associated virus vectors and neurotoxicity-lessons from preclinical and human studies. Gene Ther. 2025;2(1):60\u201373.","journal-title":"Gene Ther."},{"issue":"11","key":"84_CR18","doi-asserted-by":"publisher","first-page":"3123","DOI":"10.1016\/j.ymthe.2023.10.015","volume":"31","author":"D Duan","year":"2023","unstructured":"Duan D. Lethal immunotoxicity in high-dose systemic AAV therapy. Mol Ther. 2023;31(11):3123\u20133126.","journal-title":"Mol Ther."},{"issue":"1","key":"84_CR19","doi-asserted-by":"publisher","first-page":"78","DOI":"10.1038\/s41392-024-01780-w","volume":"9","author":"JH Wang","year":"2024","unstructured":"Wang JH, Gessler DJ, Zhan W, et al. Adeno-associated virus as a delivery vector for gene therapy of human diseases. Signal Transduct Target Ther. 2024;9(1):78.","journal-title":"Signal Transduct Target Ther."},{"key":"84_CR20","doi-asserted-by":"publisher","first-page":"303","DOI":"10.1016\/j.jconrel.2025.01.021","volume":"379","author":"N Luo","year":"2025","unstructured":"Luo N, Lin K, Cai Y, et al. An engineered adeno-associated virus mediates efficient blood\u2012brain barrier penetration with enhanced neurotropism and reduced hepatotropism. J Control Release. 2025;379:303\u2013315.","journal-title":"J Control Release."},{"key":"84_CR21","doi-asserted-by":"crossref","unstructured":"High-dose AAV gene therapy deaths. Nat Biotechnol. 2020;38(8):910.","DOI":"10.1038\/s41587-020-0642-9"},{"issue":"13","key":"84_CR22","doi-asserted-by":"publisher","first-page":"5792","DOI":"10.1167\/iovs.17-22473","volume":"58","author":"IP Seitz","year":"2017","unstructured":"Seitz IP, Michalakis S, Wilhelm B, et al. Superior Retinal Gene Transfer and Biodistribution Profile of Subretinal Versus Intravitreal Delivery of AAV8 in Nonhuman Primates. Invest Ophthalmol Vis Sci. 2017;58(13):5792\u20135801.","journal-title":"Invest Ophthalmol Vis Sci."},{"key":"84_CR23","doi-asserted-by":"publisher","first-page":"42","DOI":"10.3389\/fnana.2014.00042","volume":"8","author":"DJ Schuster","year":"2014","unstructured":"Schuster DJ, Dykstra JA, Riedl MS, et al. Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse. Front Neuroanat. 2014;8:42.","journal-title":"Front Neuroanat."},{"issue":"1","key":"84_CR24","doi-asserted-by":"publisher","first-page":"53","DOI":"10.1038\/mt.2014.180","volume":"23","author":"JI Ayers","year":"2015","unstructured":"Ayers JI, Fromholt S, Sinyavskaya O, et al. Widespread and efficient transduction of spinal cord and brain following neonatal AAV injection and potential disease modifying effect in ALS mice. Mol Ther. 2015;23(1):53\u201362.","journal-title":"Mol Ther."},{"key":"84_CR25","doi-asserted-by":"publisher","DOI":"10.1016\/j.brainres.2020.146832","volume":"1739","author":"RM Bailey","year":"2020","unstructured":"Bailey RM, Rozenberg A, Gray SJ. Comparison of high-dose intracisterna magna and lumbar puncture intrathecal delivery of AAV9 in mice to treat neuropathies. Brain Res. 2020;1739:146832.","journal-title":"Brain Res."},{"issue":"7","key":"84_CR26","doi-asserted-by":"publisher","first-page":"576","DOI":"10.1089\/hum.2017.187","volume":"28","author":"LR Belur","year":"2017","unstructured":"Belur LR, Temme A, Podetz-Pedersen KM, et al. Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I. Hum Gene Ther. 2017;28(7):576\u2013587.","journal-title":"Hum Gene Ther."},{"issue":"12","key":"84_CR27","doi-asserted-by":"publisher","DOI":"10.1002\/adhm.202303546","volume":"13","author":"Y Kurashina","year":"2024","unstructured":"Kurashina Y, Kurihara S, Kubota T, et al. Adeno-Associated Virus-Encapsulated Alginate Microspheres Loaded in Collagen Gel Carriers for Localized Gene Transfer. Adv Healthc Mater. 2024;13(12):e2303546.","journal-title":"Adv Healthc Mater."},{"issue":"3","key":"84_CR28","doi-asserted-by":"publisher","first-page":"502","DOI":"10.1038\/mt.2009.305","volume":"18","author":"CS Mah","year":"2010","unstructured":"Mah CS, Falk DJ, Germain SA, et al. Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established disease. Mol Ther. 2010;18(3):502\u2013510.","journal-title":"Mol Ther."},{"key":"84_CR29","doi-asserted-by":"crossref","unstructured":"Shi H, Xue T, Yang Y, et al. Microneedle-mediated gene delivery for the treatment of ischemic myocardial disease. Sci Adv. 2020;6(25):eaaz3621.","DOI":"10.1126\/sciadv.aaz3621"},{"key":"84_CR30","doi-asserted-by":"publisher","first-page":"610","DOI":"10.1016\/j.jconrel.2021.08.057","volume":"338","author":"Y Liu","year":"2021","unstructured":"Liu Y, Long L, Zhang F, et al. Microneedle-mediated vascular endothelial growth factor delivery promotes angiogenesis and functional recovery after stroke. J Control Release. 2021;338:610\u2013622.","journal-title":"J Control Release."},{"issue":"3","key":"84_CR31","doi-asserted-by":"publisher","first-page":"463","DOI":"10.1038\/mt.2008.269","volume":"17","author":"H Petrs-Silva","year":"2009","unstructured":"Petrs-Silva H, Dinculescu A, Li Q, et al. High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors. Mol Ther. 2009;17(3):463\u2013471.","journal-title":"Mol Ther."},{"issue":"16","key":"84_CR32","doi-asserted-by":"publisher","first-page":"8225","DOI":"10.1128\/JVI.76.16.8225-8235.2002","volume":"76","author":"DF Lackner","year":"2002","unstructured":"Lackner DF, Muzyczka N. Studies of the mechanism of transactivation of the adeno-associated virus p19 promoter by Rep protein. J Virol. 2002;76(16):8225\u20138235.","journal-title":"J Virol."},{"issue":"1","key":"84_CR33","doi-asserted-by":"publisher","first-page":"1642","DOI":"10.1038\/s41467-021-21935-5","volume":"12","author":"TP Worner","year":"2021","unstructured":"Worner TP, Bennett A, Habka S, et al. Adeno-associated virus capsid assembly is divergent and stochastic. Nat Commun. 2021;12(1):1642.","journal-title":"Nat Commun."},{"issue":"12","key":"84_CR34","doi-asserted-by":"publisher","first-page":"3601","DOI":"10.1016\/j.ymthe.2022.07.003","volume":"30","author":"L Rode","year":"2022","unstructured":"Rode L, Bar C, Gross S, et al. AAV capsid engineering identified two novel variants with improved in vivo tropism for cardiomyocytes. Mol Ther. 2022;30(12):3601\u20133618.","journal-title":"Mol Ther."},{"key":"84_CR35","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2023.101143","volume":"31","author":"MD Hoffmann","year":"2023","unstructured":"Hoffmann MD, Zdechlik AC, He Y, et al. Multiparametric domain insertional profiling of adeno-associated virus VP1. Mol Ther Methods Clin Dev. 2023;31:101143.","journal-title":"Methods & clinical development."},{"issue":"6","key":"84_CR36","doi-asserted-by":"publisher","first-page":"1817","DOI":"10.1016\/j.celrep.2018.04.026","volume":"23","author":"AC Maurer","year":"2018","unstructured":"Maurer AC, Pacouret S, Cepeda DA, et al. The Assembly Activating Protein Promotes Stability and Interactions between AAV's Viral Proteins to Nucleate Capsid Assembly. Cell Rep. 2018;23(6):1817\u20131830.","journal-title":"Cell Rep."},{"issue":"22","key":"84_CR37","doi-asserted-by":"publisher","first-page":"10220","DOI":"10.1073\/pnas.1001673107","volume":"107","author":"F Sonntag","year":"2010","unstructured":"Sonntag F, Schmidt K, Kleinschmidt JA. A viral assembly factor promotes AAV2 capsid formation in the nucleolus. Proc Natl Acad Sci U S A. 2010;107(22):10220\u201310225.","journal-title":"Proc Natl Acad Sci U S A."},{"issue":"2","key":"84_CR38","doi-asserted-by":"publisher","first-page":"340","DOI":"10.1016\/j.ymthe.2023.12.015","volume":"32","author":"ACJ Schieferecke","year":"2024","unstructured":"Schieferecke AC J, Lee H, Chen A, et al. Evolving membrane-associated accessory protein variants for improved adeno-associated virus production. Mol Ther. 2024,32(2):340\u2013351.","journal-title":"Mol Ther."},{"issue":"3\u20134","key":"84_CR39","doi-asserted-by":"publisher","first-page":"151","DOI":"10.1089\/hum.2019.274","volume":"31","author":"LF Earley","year":"2020","unstructured":"Earley LF, Conatser LM, Lue VM, et al. Adeno-Associated Virus Serotype-Specific Inverted Terminal Repeat Sequence Role in Vector Transgene Expression. Hum Gene Ther. 2020;31(3-4):151\u2013162.","journal-title":"Hum Gene Ther."},{"key":"84_CR40","doi-asserted-by":"publisher","first-page":"1106085","DOI":"10.3389\/fmed.2023.1106085","volume":"10","author":"EM Shitik","year":"2023","unstructured":"Shitik EM, Shalik IK, Yudkin DV. AAV- based vector improvements unrelated to capsid protein modification. Front Med (Lausanne). 2023;10:1106085.","journal-title":"Front Med (Lausanne)."},{"key":"84_CR41","doi-asserted-by":"crossref","unstructured":"Issa SS, Shaimardanova AA, Solovyeva VV, et al. Various AAV Serotypes and Their Applications in Gene Therapy: An Overview. Cells. 2023;12(5).","DOI":"10.3390\/cells12050785"},{"issue":"7","key":"84_CR42","doi-asserted-by":"publisher","first-page":"e02013","DOI":"10.1128\/JVI.02013-18","volume":"93","author":"AC Maurer","year":"2019","unstructured":"Maurer AC, Cepeda Diaz AK, Vandenberghe LH, et al. Residues on Adeno-associated Virus Capsid Lumen Dictate Interactions and Compatibility with the Assembly Activating Protein. J Virol. 2019;93(7):e02013-18.","journal-title":"J Virol."},{"issue":"1","key":"84_CR43","doi-asserted-by":"publisher","first-page":"62","DOI":"10.1186\/s43141-023-00518-5","volume":"21","author":"YS Xiang","year":"2023","unstructured":"Xiang YS, Hao GG. Biophysical characterization of adeno-associated virus capsid through the viral transduction life cycle. J Genet Eng Biotechnol. 2023;21(1):62.","journal-title":"J Genet Eng Biotechnol."},{"key":"84_CR44","doi-asserted-by":"crossref","unstructured":"Xie Y, Butler M. N-glycomic profiling of capsid proteins from Adeno-Associated Virus serotypes. Glycobiology. 2024;34(1).","DOI":"10.1093\/glycob\/cwad074"},{"key":"84_CR45","doi-asserted-by":"publisher","first-page":"107","DOI":"10.1016\/j.virol.2021.08.010","volume":"563","author":"MY Chen","year":"2021","unstructured":"Chen MY, Chen W, Tong J, et al. N-terminal serine\/threonine motif has diverse and important effects on behavior of multiple AAV serotypes. Virology. 2021;563:107\u2013115.","journal-title":"Virology."},{"issue":"1","key":"84_CR46","doi-asserted-by":"crossref","first-page":"3319","DOI":"10.1038\/s41467-023-39144-7","volume":"14","author":"X Chen","year":"2023","unstructured":"Chen X, Wolfe DA, Bindu DS, et al. Functional gene delivery to and across brain vasculature of systemic AAVs with endothelial-specific tropism in rodents and broad tropism in primates. Nat Commun. 2023;14(1):3319\u20133345.","journal-title":"Nature communications."},{"issue":"5","key":"84_CR47","doi-asserted-by":"publisher","first-page":"2991","DOI":"10.1128\/JVI.03371-13","volume":"88","author":"M Mietzsch","year":"2014","unstructured":"Mietzsch M, Broecker F, Reinhardt A, et al. Differential adeno-associated virus serotype-specific interaction patterns with synthetic heparins and other glycans. J Virol. 2014;88(5):2991\u20133003.","journal-title":"J Virol."},{"key":"84_CR48","doi-asserted-by":"publisher","first-page":"124","DOI":"10.1016\/j.coviro.2017.06.003","volume":"24","author":"S Pillay","year":"2017","unstructured":"Pillay S, Carette JE. Host determinants of adeno-associated viral vector entry. Curr Opin Virol. 2017;24:124\u2013131.","journal-title":"Curr Opin Virol."},{"key":"84_CR49","doi-asserted-by":"publisher","first-page":"214","DOI":"10.1016\/j.addr.2021.01.017","volume":"170","author":"EA Chowdhury","year":"2021","unstructured":"Chowdhury EA, Meno-Tetang G, Chang HY, et al. Current progress and limitations of AAV mediated delivery of protein therapeutic genes and the importance of developing quantitative pharmacokinetic\/pharmacodynamic (PK\/PD) models. Adv Drug Deliv Rev. 2021;170:214\u2013237.","journal-title":"Adv Drug Deliv Rev."},{"key":"84_CR50","doi-asserted-by":"crossref","unstructured":"Pillay S, Zou W, Cheng F, et al. Adeno-associated Virus (AAV) Serotypes Have Distinctive Interactions with Domains of the Cellular AAV Receptor. J Virol. 2017;91(18).","DOI":"10.1128\/JVI.00391-17"},{"issue":"1","key":"84_CR51","doi-asserted-by":"publisher","first-page":"3760","DOI":"10.1038\/s41467-019-11668-x","volume":"10","author":"R Zhang","year":"2019","unstructured":"Zhang R, Xu G, Cao L, et al. Divergent engagements between adeno-associated viruses with their cellular receptor AAVR. Nat Commun. 2019;10(1):3760.","journal-title":"Nat Commun."},{"issue":"24","key":"84_CR52","doi-asserted-by":"publisher","DOI":"10.1128\/jvi.01484-22","volume":"96","author":"EE Large","year":"2022","unstructured":"Large EE, Silveria MA, Weerakoon O, et al. Cross-Species Permissivity: Structure of a Goat Adeno-Associated Virus and Its Complex with the Human Receptor AAVR. J Virol. 2022;96(24):e148422.","journal-title":"J Virol."},{"key":"84_CR53","doi-asserted-by":"crossref","unstructured":"Meyer NL, Hu G, Davulcu O, et al. Structure of the gene therapy vector, adeno-associated virus with its cell receptor, AAVR. eLife. 2019;8.","DOI":"10.7554\/eLife.44707"},{"issue":"7","key":"84_CR54","doi-asserted-by":"publisher","first-page":"1336","DOI":"10.3390\/v13071336","volume":"13","author":"EE Large","year":"2021","unstructured":"Large EE, Silveria MA, Zane GM, et al. Adeno-Associated Virus (AAV) Gene Delivery: Dissecting Molecular Interactions upon Cell Entry. Viruses. 2021;13(7):1336.","journal-title":"Viruses."},{"issue":"1","key":"84_CR55","doi-asserted-by":"publisher","first-page":"3792","DOI":"10.1038\/s41467-023-39554-7","volume":"14","author":"Z Han","year":"2023","unstructured":"Han Z, Luo N, Ma W, et al. AAV11 enables efficient retrograde targeting of projection neurons and enhances astrocyte-directed transduction. Nat Commun. 2023;14(1):3792.","journal-title":"Nat Commun."},{"key":"84_CR56","doi-asserted-by":"crossref","unstructured":"Han Z, Luo N, Wu Y, et al. AAV13 Enables Precise Targeting of Local Neural Populations. Int J Mol Sci. 2022;23(21).","DOI":"10.3390\/ijms232112806"},{"issue":"4","key":"84_CR57","first-page":"781","volume":"45","author":"N Luo","year":"2024","unstructured":"Luo N, Cai Y, Han Z, et al. Engineered AAV13 variants with enhanced transduction and confined spread. Zool Res. 2024;45(4):781\u2013790.","journal-title":"Zool Res."},{"key":"84_CR58","doi-asserted-by":"publisher","first-page":"16","DOI":"10.1016\/j.coviro.2016.07.006","volume":"21","author":"D Duan","year":"2016","unstructured":"Duan D. Systemic delivery of adeno-associated viral vectors. Curr Opin Virol. 2016;21:16\u201325.","journal-title":"Curr Opin Virol."},{"issue":"25","key":"84_CR59","doi-asserted-by":"publisher","first-page":"2357","DOI":"10.1056\/NEJMoa1108046","volume":"365","author":"AC Nathwani","year":"2011","unstructured":"Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med. 2011;365(25):2357\u20132365.","journal-title":"N Engl J Med."},{"key":"84_CR60","doi-asserted-by":"publisher","first-page":"491","DOI":"10.1182\/blood-2018-99-118334","volume":"132","author":"AC Nathwani","year":"2018","unstructured":"Nathwani AC, Reiss U, Tuddenham E, et al. Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing \u201cEmpty Viral Particles\u201d on Safety and Efficacy of Gene Transfer. Blood. 2018;132:491.","journal-title":"Blood."},{"issue":"26","key":"84_CR61","doi-asserted-by":"publisher","first-page":"2519","DOI":"10.1056\/NEJMoa1708483","volume":"377","author":"S Rangarajan","year":"2017","unstructured":"Rangarajan S, Walsh L, Lester W, et al. AAV5-Factor VIII Gene Transfer in Severe Hemophilia A. N Engl J Med. 2017;377(26):2519\u20132530.","journal-title":"N Engl J Med."},{"issue":"1","key":"84_CR62","doi-asserted-by":"publisher","first-page":"29","DOI":"10.1056\/NEJMoa1908490","volume":"382","author":"KJ Pasi","year":"2020","unstructured":"Pasi KJ, Rangarajan S, Mitchell N, et al. Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A. N Engl J Med. 2020;382(1):29\u201340.","journal-title":"N Engl J Med."},{"issue":"8","key":"84_CR63","doi-asserted-by":"publisher","first-page":"828","DOI":"10.1038\/nm1085","volume":"10","author":"P Gregorevic","year":"2004","unstructured":"Gregorevic P, Blankinship MJ, Allen JM, et al. Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat Med. 2004;10(8):828\u2013834.","journal-title":"Nat Med."},{"issue":"3","key":"84_CR64","doi-asserted-by":"publisher","first-page":"321","DOI":"10.1038\/nbt1073","volume":"23","author":"Z Wang","year":"2005","unstructured":"Wang Z, Zhu T, Qiao C, et al. Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart. Nat Biotechnol. 2005;23(3):321\u2013328.","journal-title":"Nat Biotechnol."},{"issue":"1","key":"84_CR65","doi-asserted-by":"publisher","first-page":"45","DOI":"10.1016\/j.ymthe.2006.03.014","volume":"14","author":"K Inagaki","year":"2006","unstructured":"Inagaki K, Fuess S, Storm TA, et al. Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. Mol Ther. 2006;14(1):45\u201353.","journal-title":"Mol Ther."},{"issue":"1","key":"84_CR66","doi-asserted-by":"publisher","first-page":"13","DOI":"10.1038\/s41572-021-00248-3","volume":"7","author":"D Duan","year":"2021","unstructured":"Duan D, Goemans N, Takeda S, et al. Duchenne muscular dystrophy. Nat Rev Dis Primers. 2021;7(1):13.","journal-title":"Nat Rev Dis Primers."},{"issue":"1","key":"84_CR67","doi-asserted-by":"publisher","first-page":"59","DOI":"10.1038\/nbt.1515","volume":"27","author":"KD Foust","year":"2009","unstructured":"Foust KD, Nurre E, Montgomery CL, et al. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol. 2009;27(1):59\u201365.","journal-title":"Nat Biotechnol."},{"key":"84_CR68","doi-asserted-by":"publisher","first-page":"129","DOI":"10.1016\/j.jconrel.2021.03.029","volume":"333","author":"W Chen","year":"2021","unstructured":"Chen W, Yao S, Wan J, et al. BBB-crossing adeno-associated virus vector: An excellent gene delivery tool for CNS disease treatment. J Control Release. 2021;333:129\u2013138.","journal-title":"J Control Release."},{"key":"84_CR69","doi-asserted-by":"publisher","first-page":"667","DOI":"10.1016\/j.jconrel.2022.09.048","volume":"351","author":"RH Kofoed","year":"2022","unstructured":"Kofoed RH, Dibia CL, Noseworthy K, et al. Efficacy of gene delivery to the brain using AAV and ultrasound depends on serotypes and brain areas. J Control Release. 2022;351:667\u2013680.","journal-title":"J Control Release."},{"key":"84_CR70","doi-asserted-by":"publisher","first-page":"331","DOI":"10.1016\/j.omtm.2022.07.010","volume":"26","author":"D Goertsen","year":"2022","unstructured":"Goertsen D, Goeden N, Flytzanis NC, et al. Targeting the lung epithelium after intravenous delivery by directed evolution of underexplored sites on the AAV capsid. Mol Ther Methods Clin Dev. 2022;26:331\u2013342.","journal-title":"Mol Ther Methods Clin Dev."},{"issue":"7","key":"84_CR71","doi-asserted-by":"publisher","first-page":"451","DOI":"10.1038\/s41581-023-00702-3","volume":"19","author":"JL Peek","year":"2023","unstructured":"Peek JL, Wilson MH. Cell and gene therapy for kidney disease. Nat Rev Nephrol. 2023;19(7):451\u2013462.","journal-title":"Nat Rev Nephrol."},{"issue":"17","key":"84_CR72","doi-asserted-by":"publisher","DOI":"10.1172\/JCI174722","volume":"134","author":"G Wu","year":"2024","unstructured":"Wu G, Liu S, Hagenstein J, et al. Adeno-associated virus-based gene therapy treats inflammatory kidney disease in mice. J Clin Invest. 2024;134(17):e174722.","journal-title":"J Clin Invest."},{"issue":"1","key":"84_CR73","doi-asserted-by":"publisher","first-page":"9609","DOI":"10.1038\/s41598-017-09805-x","volume":"7","author":"SB Shibata","year":"2017","unstructured":"Shibata SB, Yoshimura H, Ranum PT, et al. Intravenous rAAV2\/9 injection for murine cochlear gene delivery. Sci Rep. 2017;7(1):9609.","journal-title":"Sci Rep."},{"issue":"8","key":"84_CR74","doi-asserted-by":"publisher","DOI":"10.15252\/emmm.201809431","volume":"10","author":"CH Sponton","year":"2018","unstructured":"Sponton CH, Kajimura S. AAV-mediated gene therapy as a strategy to fight obesity and metabolic diseases. EMBO Mol Med. 2018;10(8):e9431.","journal-title":"EMBO Mol Med."},{"key":"84_CR75","doi-asserted-by":"publisher","first-page":"287","DOI":"10.1016\/j.jconrel.2016.01.001","volume":"240","author":"JL Santiago-Ortiz","year":"2016","unstructured":"Santiago-Ortiz JL, Schaffer DV. Adeno-associated virus (AAV) vectors in cancer gene therapy. J Control Release. 2016;240:287\u2013301.","journal-title":"J Control Release."},{"issue":"4","key":"84_CR76","doi-asserted-by":"publisher","first-page":"450","DOI":"10.1038\/gt.2012.101","volume":"20","author":"SJ Gray","year":"2013","unstructured":"Gray SJ, Nagabhushan KS, McCown TJ, et al. Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in nonhuman primates. Gene Ther. 2013;20(4):450\u2013459.","journal-title":"Gene Ther."},{"key":"84_CR77","doi-asserted-by":"publisher","DOI":"10.1016\/j.preteyeres.2020.100915","volume":"83","author":"K Bucher","year":"2021","unstructured":"Bucher K, Rodriguez-Bocanegra E, Dauletbekov D, et al. Immune responses to retinal gene therapy using adeno-associated viral vectors - Implications for treatment success and safety. Prog Retin Eye Res. 2021;83:100915.","journal-title":"Prog Retin Eye Res."},{"issue":"11","key":"84_CR78","doi-asserted-by":"publisher","first-page":"4901","DOI":"10.1172\/JCI129085","volume":"129","author":"K Ding","year":"2019","unstructured":"Ding K, Shen J, Hafiz Z, et al. AAV8-vectored suprachoroidal gene transfer produces widespread ocular transgene expression. J Clin Invest. 2019;129(11):4901\u20134911.","journal-title":"J Clin Invest."},{"issue":"6","key":"84_CR79","doi-asserted-by":"publisher","first-page":"402","DOI":"10.1038\/s41434-018-0035-6","volume":"25","author":"L Song","year":"2018","unstructured":"Song L, Llanga T, Conatser LM, et al. Serotype survey of AAV gene delivery via subconjunctival injection in mice. Gene Ther. 2018;25(6):402\u2013414.","journal-title":"Gene Ther."},{"key":"84_CR80","doi-asserted-by":"publisher","first-page":"143","DOI":"10.1016\/j.omtm.2019.11.017","volume":"17","author":"J Tan","year":"2020","unstructured":"Tan J, Wang X, Cai S, et al. C3 Transferase-Expressing scAAV2 Transduces Ocular Anterior Segment Tissues and Lowers Intraocular Pressure in Mouse and Monkey. Mol Ther Methods Clin Dev. 2020;17:143\u2013155.","journal-title":"Mol Ther Methods Clin Dev."},{"issue":"9","key":"84_CR81","doi-asserted-by":"publisher","first-page":"1308","DOI":"10.1038\/eye.2017.158","volume":"31","author":"K Xue","year":"2017","unstructured":"Xue K, Groppe M, Salvetti AP, et al. Technique of retinal gene therapy: delivery of viral vector into the subretinal space. Eye (Lond). 2017;31(9):1308\u20131316.","journal-title":"Eye (Lond)."},{"key":"84_CR82","first-page":"1760","volume":"14","author":"Q Li","year":"2008","unstructured":"Li Q, Miller R, Han PY, et al. Intraocular route of AAV2 vector administration defines humoral immune response and therapeutic potential. Mol Vis. 2008;14:1760\u20131769.","journal-title":"Mol Vis."},{"key":"84_CR83","doi-asserted-by":"publisher","first-page":"458","DOI":"10.1016\/j.jconrel.2023.01.067","volume":"355","author":"L Kang","year":"2023","unstructured":"Kang L, Jin S, Wang J, et al. AAV vectors applied to the treatment of CNS disorders: Clinical status and challenges. J Control Release. 2023;355:458\u2013473.","journal-title":"J Control Release."},{"issue":"12","key":"84_CR84","doi-asserted-by":"publisher","first-page":"1164","DOI":"10.1016\/S1474-4422(10)70254-4","volume":"9","author":"WJ Marks","year":"2010","unstructured":"Marks WJ, Bartus RT, Siffert J, et al. Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomized, controlled trial. Lancet Neurol. 2010;9(12):1164\u20131172.","journal-title":"Lancet Neurol."},{"issue":"5","key":"84_CR85","doi-asserted-by":"publisher","first-page":"704","DOI":"10.1002\/ana.25450","volume":"85","author":"CW Christine","year":"2019","unstructured":"Christine CW, Bankiewicz KS, Van Laar AD, et al. Magnetic resonance imaging-guided phase 1 trial of putaminal AADC gene therapy for Parkinson's disease. Ann Neurol. 2019;85(5):704\u2013714.","journal-title":"Ann Neurol."},{"issue":"1","key":"84_CR86","doi-asserted-by":"publisher","first-page":"2","DOI":"10.1006\/exnr.2000.7408","volume":"164","author":"KS Bankiewicz","year":"2000","unstructured":"Bankiewicz KS, Eberling JL, Kohutnicka M, et al. Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach. Exp Neurol. 2000;164(1):2\u201314.","journal-title":"Exp Neurol."},{"issue":"24","key":"84_CR87","doi-asserted-by":"publisher","first-page":"12382","DOI":"10.1128\/JVI.75.24.12382-12392.2001","volume":"75","author":"MA Passini","year":"2001","unstructured":"Passini MA, Wolfe JH. Widespread gene delivery and structure-specific patterns of expression in the brain after intraventricular injections of neonatal mice with an adeno-associated virus vector. J Virol. 2001;75(24):12382\u201312392.","journal-title":"J Virol."},{"key":"84_CR88","first-page":"51863","volume":"91","author":"JY Kim","year":"2014","unstructured":"Kim JY, Grunke SD, Levites Y, et al. Intracerebroventricular viral injection of the neonatal mouse brain for persistent and widespread neuronal transduction. J Vis Exp. 2014; (91):51863.","journal-title":"J Vis Exp."},{"issue":"6","key":"84_CR89","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0067680","volume":"8","author":"P Chakrabarty","year":"2013","unstructured":"Chakrabarty P, Rosario A, Cruz P, et al. Capsid serotype and timing of injection determines AAV transduction in the neonatal mice brain. PLoS One. 2013;8(6):e67680.","journal-title":"PLoS One."},{"key":"84_CR90","doi-asserted-by":"publisher","DOI":"10.3389\/fnmol.2021.618360","volume":"14","author":"LR Belur","year":"2021","unstructured":"Belur LR, Romero M, Lee J, et al. Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I. Front Mol Neurosci. 2021;14:618360.","journal-title":"Front Mol Neurosci."},{"issue":"1","key":"84_CR91","doi-asserted-by":"publisher","first-page":"76","DOI":"10.1186\/s12935-021-01776-4","volume":"21","author":"X Xu","year":"2021","unstructured":"Xu X, Chen W, Zhu W, et al. Adeno-associated virus (AAV)-based gene therapy for glioblastoma. Cancer Cell Int. 2021;21(1):76.","journal-title":"Cancer Cell Int."},{"issue":"1","key":"84_CR92","doi-asserted-by":"publisher","first-page":"66","DOI":"10.1186\/s40478-017-0464-2","volume":"5","author":"J Hordeaux","year":"2017","unstructured":"Hordeaux J, Dubreil L, Robveille C, et al. Long-term neurologic and cardiac correction by intrathecal gene therapy in Pompe disease. Acta Neuropathol Commun. 2017;5(1):66.","journal-title":"Acta Neuropathol Commun."},{"issue":"11","key":"84_CR93","doi-asserted-by":"publisher","first-page":"3277","DOI":"10.1016\/j.ymthe.2023.09.018","volume":"31","author":"W Cheng","year":"2023","unstructured":"Cheng W, Huang J, Fu XQ, et al. Intrathecal delivery of AAV-NDNF ameliorates disease progression of ALS mice. Mol Ther. 2023;31(11):3277\u20133289.","journal-title":"Mol Ther."},{"key":"84_CR94","doi-asserted-by":"publisher","DOI":"10.1016\/j.expneurol.2021.113945","volume":"348","author":"JD Sydney-Smith","year":"2022","unstructured":"Sydney-Smith JD, Spejo AB, Warren PM, et al. Peripherally delivered Adeno-associated viral vectors for spinal cord injury repair. Exp Neurol. 2022;348:113945.","journal-title":"Exp Neurol."},{"issue":"2","key":"84_CR95","doi-asserted-by":"publisher","first-page":"411","DOI":"10.1016\/j.ymthe.2019.11.012","volume":"28","author":"T Taghian","year":"2020","unstructured":"Taghian T, Marosfoi MG, Puri AS, et al. A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna. Mol Ther. 2020;28(2):411\u2013421.","journal-title":"Mol Ther."},{"issue":"3","key":"84_CR96","doi-asserted-by":"publisher","first-page":"477","DOI":"10.1038\/mt.2014.210","volume":"23","author":"K Meyer","year":"2015","unstructured":"Meyer K, Ferraiuolo L, Schmelzer L, et al. Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates. Mol Ther. 2015;23(3):477\u2013487.","journal-title":"Mol Ther."},{"key":"84_CR97","doi-asserted-by":"publisher","first-page":"22404","DOI":"10.1038\/srep22404","volume":"6","author":"XC Ma","year":"2016","unstructured":"Ma XC, Liu P, Zhang XL, et al. Intranasal Delivery of Recombinant AAV Containing BDNF Fused with HA2TAT: a Potential Promising Therapy Strategy for Major Depressive Disorder. Sci Rep. 2016;6:22404.","journal-title":"Sci Rep."},{"issue":"6","key":"84_CR98","doi-asserted-by":"publisher","first-page":"2621","DOI":"10.1128\/JVI.80.6.2621-2630.2006","volume":"80","author":"D Kuck","year":"2006","unstructured":"Kuck D, Lau T, Leuchs B, et al. Intranasal vaccination with recombinant adeno-associated virus type 5 against human papillomavirus type 16 L1. J Virol. 2006;80(6):2621\u20132630.","journal-title":"J Virol."},{"issue":"7","key":"84_CR99","doi-asserted-by":"publisher","first-page":"733","DOI":"10.1089\/hum.2011.202","volume":"23","author":"K Nieto","year":"2012","unstructured":"Nieto K, Stahl-Hennig C, Leuchs B, et al. Intranasal vaccination with AAV5 and 9 vectors against human papillomavirus type 16 in rhesus macaques. Hum Gene Ther. 2012;23(7):733\u2013741.","journal-title":"Hum Gene Ther."},{"issue":"11","key":"84_CR100","doi-asserted-by":"publisher","first-page":"1349","DOI":"10.1089\/104303402760128577","volume":"13","author":"JA Wagner","year":"2002","unstructured":"Wagner JA, Nepomuceno IB, Messner AH, et al. A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies. Hum Gene Ther. 2002;13(11):1349\u20131359.","journal-title":"Hum Gene Ther."},{"issue":"1","key":"84_CR101","doi-asserted-by":"publisher","first-page":"192","DOI":"10.1038\/mt.2014.200","volume":"23","author":"JR Mendell","year":"2015","unstructured":"Mendell JR, Sahenk Z, Malik V, et al. A phase 1\/2a follistatin gene therapy trial for becker muscular dystrophy. Mol Ther. 2015;23(1):192\u2013201.","journal-title":"Mol Ther."},{"key":"84_CR102","doi-asserted-by":"publisher","first-page":"14848","DOI":"10.1038\/ncomms14848","volume":"8","author":"A Malerba","year":"2017","unstructured":"Malerba A, Klein P, Bachtarzi H, et al. PABPN1 gene therapy for oculopharyngeal muscular dystrophy. Nat Commun. 2017;8:14848.","journal-title":"Nat Commun."},{"key":"84_CR103","doi-asserted-by":"crossref","unstructured":"Pleger ST, Shan C, Ksienzyk J, et al. Cardiac AAV9-S100A1 gene therapy rescues postischemic heart failure in a preclinical large animal model. Sci Transl Med. 2011;3(92):92ra64.","DOI":"10.1126\/scitranslmed.3002097"},{"issue":"1","key":"84_CR104","doi-asserted-by":"publisher","first-page":"89","DOI":"10.1161\/CIRCULATIONAHA.108.803999","volume":"119","author":"G Rengo","year":"2009","unstructured":"Rengo G, Lymperopoulos A, Zincarelli C, et al. Myocardial adeno-associated virus serotype 6-betaARKct gene therapy improves cardiac function and normalizes the neurohormonal axis in chronic heart failure. Circulation. 2009;119(1):89\u201398.","journal-title":"Circulation."},{"issue":"1","key":"84_CR105","doi-asserted-by":"publisher","first-page":"4722","DOI":"10.1038\/s41467-021-24849-4","volume":"12","author":"RJ Chai","year":"2021","unstructured":"Chai RJ, Werner H, Li PY, et al. Disrupting the LINC complex by AAV mediated gene transduction prevents progression of Lamin induced cardiomyopathy. Nat Commun. 2021;12(1):4722.","journal-title":"Nat Commun."},{"issue":"13","key":"84_CR106","doi-asserted-by":"publisher","first-page":"1626","DOI":"10.1161\/01.CIR.0000089371.11664.27","volume":"108","author":"Y Yue","year":"2003","unstructured":"Yue Y, Li Z, Harper SQ, et al. Microdystrophin gene therapy of cardiomyopathy restores dystrophin-glycoprotein complex and improves sarcolemma integrity in the mdx mouse heart. Circulation. 2003;108(13):1626\u20131632.","journal-title":"Circulation."},{"issue":"4","key":"84_CR107","doi-asserted-by":"publisher","first-page":"234","DOI":"10.1089\/hgtb.2012.105","volume":"23","author":"H Fang","year":"2012","unstructured":"Fang H, Lai NC, Gao MH, et al. Comparison of adeno-associated virus serotypes and delivery methods for cardiac gene transfer. Hum Gene Ther Methods. 2012;23(4):234\u2013241.","journal-title":"Hum Gene Ther Methods."},{"issue":"4","key":"84_CR108","doi-asserted-by":"publisher","first-page":"382","DOI":"10.1053\/rmed.2002.1457","volume":"97","author":"DA Groneberg","year":"2003","unstructured":"Groneberg DA, Witt C, Wagner U, et al. Fundamentals of pulmonary drug delivery. Respir Med. 2003;97(4):382\u2013387.","journal-title":"Respir Med."},{"key":"84_CR109","unstructured":"Scheuch G, Siekmeier R. Novel approaches to enhance pulmonary delivery of proteins and peptides. J Physiol Pharmacol. 2007;58 Suppl 5(Pt 2):615\u2013625."},{"issue":"2","key":"84_CR110","doi-asserted-by":"publisher","first-page":"509","DOI":"10.1378\/chest.125.2.509","volume":"125","author":"RB Moss","year":"2004","unstructured":"Moss RB, Rodman D, Spencer LT, et al. Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: a multicenter, double-blind, placebo-controlled trial. Chest. 2004;125(2):509\u2013521.","journal-title":"Chest."},{"issue":"1","key":"84_CR111","doi-asserted-by":"publisher","first-page":"36","DOI":"10.1089\/hum.2014.053","volume":"26","author":"RJ MacLoughlin","year":"2015","unstructured":"MacLoughlin RJ, Higgins BD, Devaney J, et al. Aerosol-mediated delivery of AAV2\/6-IkappaBalpha attenuates lipopolysaccharide-induced acute lung injury in rats. Hum Gene Ther. 2015;26(1):36\u201346.","journal-title":"Hum Gene Ther."},{"issue":"17","key":"84_CR112","doi-asserted-by":"publisher","first-page":"2032","DOI":"10.1016\/j.jacc.2016.02.049","volume":"67","author":"J Aguero","year":"2016","unstructured":"Aguero J, Ishikawa K, Hadri L, et al. Intratracheal Gene Delivery of SERCA2a Ameliorates Chronic Post-Capillary Pulmonary Hypertension: A Large Animal Model. J Am Coll Cardiol. 2016;67(17):2032\u20132046.","journal-title":"J Am Coll Cardiol."},{"issue":"21\u201322","key":"84_CR113","first-page":"1135","volume":"34","author":"MK Yanda","year":"2023","unstructured":"Yanda MK, Zeidan A, Ciobanu C, et al. Transduction of Ferret Surface and Basal Cells of Airways, Lung, Liver, and Pancreas via Intratracheal or Intravenous Delivery of Adeno-Associated Virus 1 or 6. Hum Gene Ther. 2023;34(21\u201322):1135\u20131144.","journal-title":"Hum Gene Ther."},{"key":"84_CR114","doi-asserted-by":"crossref","unstructured":"Bikou O, Tharakan S, Yamada KP, et al. Endobronchial Aerosolized AAV1.SERCA2a Gene Therapy in a Pulmonary Hypertension Pig Model: Addressing the Lung Delivery Bottleneck. Hum Gene Ther. 2022;33(9\u201310):550\u2013559.","DOI":"10.1089\/hum.2021.274"},{"issue":"4","key":"84_CR115","doi-asserted-by":"publisher","first-page":"460","DOI":"10.1038\/gt.2012.67","volume":"20","author":"AP Dane","year":"2013","unstructured":"Dane AP, Wowro SJ, Cunningham SC, et al. Comparison of gene transfer to the murine liver following intraperitoneal and intraportal delivery of hepatotropic AAV pseudoserotypes. Gene Ther. 2013;20(4):460\u2013464.","journal-title":"Gene Ther."},{"issue":"2","key":"84_CR116","doi-asserted-by":"publisher","first-page":"318","DOI":"10.1038\/mt.2012.258","volume":"21","author":"J Mimuro","year":"2013","unstructured":"Mimuro J, Mizukami H, Hishikawa S, et al. Minimizing the inhibitory effect of neutralizing antibody for efficient gene expression in the liver with adeno-associated virus 8 vectors. Mol Ther. 2013;21(2):318\u2013323.","journal-title":"Mol Ther."},{"key":"84_CR117","doi-asserted-by":"publisher","first-page":"1028380","DOI":"10.3389\/fcimb.2022.1028380","volume":"12","author":"L Ma","year":"2022","unstructured":"Ma L, Lian J, Bai Y, et al. Adeno-associated virus vector intraperitoneal injection induces colonic mucosa and submucosa transduction and alters the diversity and composition of the fecal microbiota in rats. Front Cell Infect Microbiol. 2022;12:1028380.","journal-title":"Frontiers in cellular and infection microbiology."},{"issue":"5","key":"84_CR118","doi-asserted-by":"publisher","first-page":"356","DOI":"10.1038\/s41417-019-0108-8","volume":"27","author":"DL Yu","year":"2020","unstructured":"Yu DL, Stegelmeier AA, Chow N, et al. AAV-mediated expression of 3TSR inhibits tumor and metastatic lesion development and extends survival in a murine model of epithelial ovarian carcinoma. Cancer Gene Ther. 2020;27(5):356\u2013367.","journal-title":"Cancer Gene Ther."},{"issue":"12","key":"84_CR119","doi-asserted-by":"publisher","first-page":"1559","DOI":"10.1089\/hum.2019.127","volume":"30","author":"JD Rubin","year":"2019","unstructured":"Rubin JD, Nguyen TV, Allen KL, et al. Comparison of Gene Delivery to the Kidney by Adenovirus, Adeno-Associated Virus, and Lentiviral Vectors After Intravenous and Direct Kidney Injections. Hum Gene Ther. 2019;30(12):1559\u20131571.","journal-title":"Hum Gene Ther."},{"issue":"10441","key":"84_CR120","doi-asserted-by":"publisher","first-page":"2317","DOI":"10.1016\/S0140-6736(23)02874-X","volume":"403","author":"J Lv","year":"2024","unstructured":"Lv J, Wang H, Cheng X, et al. AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial. Lancet. 2024;403(10441):2317\u20132325.","journal-title":"Lancet."},{"issue":"11","key":"84_CR121","doi-asserted-by":"publisher","DOI":"10.1002\/advs.202306788","volume":"11","author":"J Qi","year":"2024","unstructured":"Qi J, Tan F, Zhang L, et al. AAV-Mediated Gene Therapy Restores Hearing in Patients with DFNB9 Deafness. Adv Sci (Weinh) . 2024;11(11):e2306788.","journal-title":"Adv Sci (Weinh)."},{"key":"84_CR122","doi-asserted-by":"crossref","unstructured":"Zhang S, Yang B, Shen X, et al. AAV-mediated gene therapy restores natural fertility and improves physical function in the Lhcgr-deficient mouse model of Leydig cell failure. Cell Prolif. 2024;e13680.","DOI":"10.1111\/cpr.13680"},{"issue":"1","key":"84_CR123","doi-asserted-by":"publisher","first-page":"70","DOI":"10.1186\/s12896-018-0479-1","volume":"18","author":"S Rajasekaran","year":"2018","unstructured":"Rajasekaran S, Thatte J, Periasamy J, et al. Infectivity of adeno-associated virus serotypes in mouse testis. BMC Biotechnol. 2018;18(1):70.","journal-title":"BMC Biotechnol."},{"issue":"11","key":"84_CR124","first-page":"1508","volume":"18","author":"D GuhaSarkar","year":"2016","unstructured":"GuhaSarkar D, Su Q, Gao G, et al. Systemic AAV9-IFNbeta gene delivery treats highly invasive glioblastoma. Neuro Oncol. 2016;18(11):1508\u20131518.","journal-title":"Neuro Oncol."},{"issue":"1","key":"84_CR125","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0054146","volume":"8","author":"S Watanabe","year":"2013","unstructured":"Watanabe S, Sanuki R, Ueno S, et al. Tropisms of AAV for subretinal delivery to the neonatal mouse retina and its application for in vivo rescue of developmental photoreceptor disorders. PLoS One. 2013;8(1):e54146.","journal-title":"PLoS One."},{"key":"84_CR126","doi-asserted-by":"publisher","first-page":"179","DOI":"10.1016\/j.omtm.2020.01.002","volume":"16","author":"G Yiu","year":"2020","unstructured":"Yiu G, Chung SH, Mollhoff IN, et al. Suprachoroidal and Subretinal Injections of AAV Using Transscleral Microneedles for Retinal Gene Delivery in Nonhuman Primates. Mol Ther Methods Clin Dev. 2020;16:179\u2013191.","journal-title":"Mol Ther Methods Clin Dev."},{"issue":"2","key":"84_CR127","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2024.101242","volume":"32","author":"SH Haldrup","year":"2024","unstructured":"Haldrup SH, Fabian-Jessing BK, Jakobsen TS, et al. Subretinal AAV delivery of RNAi-therapeutics targeting VEGFA reduces choroidal neovascularization in a large animal model. Mol Ther Methods Clin Dev. 2024;32(2):101242.","journal-title":"Mol Ther Methods Clin Dev."},{"issue":"10","key":"84_CR128","doi-asserted-by":"publisher","first-page":"763","DOI":"10.1038\/s41419-024-07150-2","volume":"15","author":"K Azuma","year":"2024","unstructured":"Azuma K, Suzuki T, Kobayashi K, et al. Retinal pigment epithelium-specific ablation of GPx4 in adult mice recapitulates key features of geographic atrophy in age\u2013related macular degeneration. Cell Death Dis. 2024;15(10):763.","journal-title":"Cell Death Dis."},{"issue":"1","key":"84_CR129","doi-asserted-by":"publisher","first-page":"6578","DOI":"10.1038\/s41467-023-42386-0","volume":"14","author":"LM Riedmayr","year":"2023","unstructured":"Riedmayr LM, Hinrichsmeyer KS, Thalhammer SB, et al. mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy. Nat Commun. 2023;14(1):6578.","journal-title":"Nat Commun."},{"issue":"13","key":"84_CR130","doi-asserted-by":"publisher","first-page":"5398","DOI":"10.1167\/iovs.18-24926","volume":"59","author":"S Lee","year":"2018","unstructured":"Lee S, Kim HJ, Shin OK, et al. Intravitreal Injection of AAV Expressing Soluble VEGF Receptor-1 Variant Induces Anti-VEGF Activity and Suppresses Choroidal Neovascularization. Invest Ophthalmol Vis Sci. 2018;59(13):5398\u20135407.","journal-title":"Invest Ophthalmol Vis Sci."},{"issue":"11","key":"84_CR131","doi-asserted-by":"publisher","first-page":"3308","DOI":"10.1016\/j.ymthe.2023.09.019","volume":"31","author":"X Ke","year":"2023","unstructured":"Ke X, Jiang H, Li Q, et al. Preclinical evaluation of KH631, a novel rAAV8 gene therapy product for neovascular age-related macular degeneration. Mol Ther. 2023;31(11):3308\u20133321.","journal-title":"Mol Ther."},{"issue":"1","key":"84_CR132","doi-asserted-by":"publisher","DOI":"10.1016\/j.xops.2024.100590","volume":"5","author":"GW Roddy","year":"2025","unstructured":"Roddy GW, Kohli D, Niknam P, et al. Subconjunctival Administration of an Adeno-Associated Virus Expressing Stanniocalcin-1 Provides Sustained Intraocular Pressure Reduction in Mice. Ophthalmol Sci. 2025;5(1):100590.","journal-title":"Ophthalmol Sci."},{"key":"84_CR133","doi-asserted-by":"publisher","first-page":"107","DOI":"10.1016\/j.omtm.2021.06.007","volume":"22","author":"W Su","year":"2021","unstructured":"Su W, Sun S, Tian B, et al. Efficacious, safe, and stable inhibition of corneal neovascularization by AAV\u2013vectored anti-VEGF therapeutics. Mol Ther Methods Clin Dev. 2021;22:107\u2013121.","journal-title":"Mol Ther Methods Clin Dev."},{"issue":"7","key":"84_CR134","doi-asserted-by":"publisher","first-page":"1230","DOI":"10.1093\/hmg\/ddx028","volume":"26","author":"J O'Callaghan","year":"2017","unstructured":"O'Callaghan J, Crosbie DE, Cassidy PS, et al. Therapeutic potential of AAV\u2013mediated MMP-3 secretion from corneal endothelium in treating glaucoma. Hum Mol Genet. 2017;26(7):1230\u20131246.","journal-title":"Hum Mol Genet."},{"issue":"1","key":"84_CR135","doi-asserted-by":"publisher","first-page":"19864","DOI":"10.1038\/s41598-019-56462-3","volume":"9","author":"E Crabtree","year":"2019","unstructured":"Crabtree E, Song L, Llanga T, et al. AAV-mediated expression of HLA\u2013G1\/5 reduces severity of experimental autoimmune uveitis. Sci Rep. 2019;9(1):19864.","journal-title":"Sci Rep."},{"issue":"1","key":"84_CR136","doi-asserted-by":"publisher","first-page":"437","DOI":"10.1186\/s12967-023-04208-1","volume":"21","author":"C O'Leary","year":"2023","unstructured":"O'Leary C, Forte G, Mitchell NL, et al. Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC. J Transl Med. 2023;21(1):437.","journal-title":"J Transl Med."},{"issue":"7\u20138","key":"84_CR137","doi-asserted-by":"publisher","first-page":"415","DOI":"10.1089\/hum.2019.367","volume":"31","author":"MJ Castle","year":"2020","unstructured":"Castle MJ, Baltanas FC, Kovacs I, et al. Postmortem Analysis in a Clinical Trial of AAV2-NGF Gene Therapy for Alzheimer's Disease Identifies a Need for Improved Vector Delivery. Hum Gene Ther. 2020;31(7\u20138):415\u2013422.","journal-title":"Hum Gene Ther."},{"key":"84_CR138","doi-asserted-by":"crossref","unstructured":"Morabito G, Giannelli SG, Ordazzo G, et al. AAV-PHP.B-Mediated Global-Scale Expression in the Mouse Nervous System Enables GBA1 Gene Therapy for Wide Protection from Synucleinopathy. Mol Ther. 2017;25(12):2727\u20132742.","DOI":"10.1016\/j.ymthe.2017.08.004"},{"issue":"5","key":"84_CR139","doi-asserted-by":"publisher","first-page":"579","DOI":"10.3340\/jkns.2019.0182","volume":"63","author":"KH So","year":"2020","unstructured":"So KH, Choi JH, Islam J, et al. An Optimization of AAV\u201382Q\u2013Delivered Rat Model of Huntington's Disease. J Korean Neurosurg Soc. 2020;63(5):579\u2013589.","journal-title":"J Korean Neurosurg Soc."},{"issue":"1","key":"84_CR140","doi-asserted-by":"publisher","first-page":"162","DOI":"10.1016\/j.ymthe.2020.09.033","volume":"29","author":"TB Johnson","year":"2021","unstructured":"Johnson TB, White KA, Brudvig JJ, et al. AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease. Mol Ther. 2021;29(1):162\u2013175.","journal-title":"Molecular therapy."},{"issue":"5","key":"84_CR141","doi-asserted-by":"publisher","first-page":"682","DOI":"10.1016\/j.ymthe.2004.02.016","volume":"9","author":"AP Kells","year":"2004","unstructured":"Kells AP, Fong DM, Dragunow M, et al. AAV-mediated gene delivery of BDNF or GDNF is neuroprotective in a model of Huntington disease. Mol Ther. 2004;9(5):682\u2013688.","journal-title":"Mol Ther."},{"issue":"2","key":"84_CR142","doi-asserted-by":"publisher","first-page":"180","DOI":"10.1002\/1878-0261.12020","volume":"11","author":"D GuhaSarkar","year":"2017","unstructured":"GuhaSarkar D, Neiswender J, Su Q, et al. Intracranial AAV-IFN-beta gene therapy eliminates invasive xenograft glioblastoma and improves survival in orthotopic syngeneic murine model. Mol Oncol. 2017;11(2):180\u2013193.","journal-title":"Mol Oncol."},{"issue":"594","key":"84_CR143","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1126\/scitranslmed.aaw1564","volume":"13","author":"J Ng","year":"2021","unstructured":"Ng J, Barral S, De La Fuente Barrigon C, et al. Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism. Sci Transl Med. 2021;13(594):1.","journal-title":"Science translational medicine."},{"key":"84_CR144","doi-asserted-by":"publisher","first-page":"73","DOI":"10.1016\/j.neulet.2014.05.044","volume":"576","author":"JR McLean","year":"2014","unstructured":"McLean JR, Smith GA, Rocha EM, et al. Widespread neuron-specific transgene expression in brain and spinal cord following synapsin promoter-driven AAV9 neonatal intracerebroventricular injection. Neurosci Lett. 2014;576:73\u201378.","journal-title":"Neurosci Lett."},{"issue":"7","key":"84_CR145","doi-asserted-by":"publisher","first-page":"619","DOI":"10.1089\/hum.2014.011","volume":"25","author":"MA Passini","year":"2014","unstructured":"Passini MA, Bu J, Richards AM, et al. Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy. Hum Gene Ther. 2014;25(7):619\u2013630.","journal-title":"Hum Gene Ther."},{"issue":"2","key":"84_CR146","doi-asserted-by":"publisher","first-page":"282","DOI":"10.1038\/mt.2012.261","volume":"21","author":"S Benkhelifa-Ziyyat","year":"2013","unstructured":"Benkhelifa-Ziyyat S, Besse A, Roda M, et al. Intramuscular scAAV9-SMN Injection Mediates Widespread Gene Delivery to the Spinal Cord and Decreases Disease Severity in SMA Mice. Mol Ther. 2013;21(2):282\u2013290.","journal-title":"Molecular therapy."},{"key":"84_CR147","doi-asserted-by":"crossref","unstructured":"Sabatino DE, Arruda VR. Muscle Gene Therapy for Hemophilia. J Genet Syndr Gene Ther. 2012;Suppl 1:S1\u2013010.","DOI":"10.4172\/2157-7412.S1-010"},{"issue":"4","key":"84_CR148","doi-asserted-by":"publisher","first-page":"189","DOI":"10.1089\/hgtb.2017.192.R3","volume":"29","author":"L Chansel-Debordeaux","year":"2018","unstructured":"Chansel-Debordeaux L, Bourdenx M, Dutheil N, et al. Systemic Gene Delivery by Single-Dose Intracardiac Administration of scAAV2\/9 and scAAV2\/rh10 Variants in Newborn Rats. Hum Gene Ther Methods. 2018;29(4):189\u2013199.","journal-title":"Hum Gene Ther Methods."},{"issue":"19","key":"84_CR149","doi-asserted-by":"publisher","DOI":"10.1111\/jcmm.70084","volume":"28","author":"P Zhong","year":"2024","unstructured":"Zhong P, Yang S, Fang C, et al. AAV9\u2010mediated CIRP gene transfer protects against cardiac dysfunction and remodeling in a rat model of myocardial infarction. J Cell Mol Med. 2024;28(19):e70084.","journal-title":"J Cell Mol Med."},{"key":"84_CR150","doi-asserted-by":"crossref","unstructured":"De BP, Heguy A, Hackett NR, et al. High levels of persistent expression of alpha1-antitrypsin mediated by the nonhuman primate serotype rh.10 adeno\u2013associated virus despite preexisting immunity to common human adeno-associated viruses. Mol Ther. 2006;13(1):67\u201376.","DOI":"10.1016\/j.ymthe.2005.09.003"},{"issue":"3","key":"84_CR151","doi-asserted-by":"publisher","first-page":"296","DOI":"10.1038\/gt.2015.114","volume":"23","author":"M Merentie","year":"2016","unstructured":"Merentie M, Lottonen-Raikaslehto L, Parviainen V, et al. Efficacy and safety of myocardial gene transfer of adenovirus, adeno-associated virus and lentiviral vectors in the mouse heart. Gene Ther. 2016;23(3):296\u2013305.","journal-title":"Gene therapy."},{"issue":"2","key":"84_CR152","doi-asserted-by":"publisher","first-page":"201","DOI":"10.1161\/01.CIR.99.2.201","volume":"99","author":"EC Svensson","year":"1999","unstructured":"Svensson EC, Marshall DJ, Woodard K, et al. Efficient and stable transduction of cardiomyocytes after intramyocardial injection or intracoronary perfusion with recombinant adeno-associated virus vectors. Circulation. 1999;99(2):201\u2013205.","journal-title":"Circulation."},{"key":"84_CR153","doi-asserted-by":"crossref","unstructured":"Wang J, Jonker T, Cervera I Barea A, et al. Adeno-associated virus (AAV) 6 provides efficient gene transfer upon intramyocardial injection in vivo. Cardiovasc Res. 2024;120(Suppl 1): cvae088.073.","DOI":"10.1093\/cvr\/cvae088.073"},{"issue":"5","key":"84_CR154","doi-asserted-by":"publisher","first-page":"405","DOI":"10.1161\/CIRCULATIONAHA.122.063759","volume":"148","author":"X Li","year":"2023","unstructured":"Li X, La Salvia S, Liang Y, et al. Extracellular Vesicle-Encapsulated Adeno-Associated Viruses for Therapeutic Gene Delivery to the Heart. Circulation. 2023;148(5):405\u2013425.","journal-title":"Circulation."}],"container-title":["Current Medical Science"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s11596-025-00084-6.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s11596-025-00084-6\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s11596-025-00084-6.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,9,25]],"date-time":"2025-09-25T04:02:56Z","timestamp":1758772976000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s11596-025-00084-6"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,7,15]]},"references-count":154,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2025,8]]}},"alternative-id":["84"],"URL":"https:\/\/doi.org\/10.1007\/s11596-025-00084-6","relation":{},"ISSN":["2096-5230","2523-899X"],"issn-type":[{"value":"2096-5230","type":"print"},{"value":"2523-899X","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,7,15]]},"assertion":[{"value":"2 February 2025","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"11 June 2025","order":2,"name":"revised","label":"Revised","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"16 June 2025","order":3,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"15 July 2025","order":4,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"The authors have declared that no competing interests exist.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of Interest"}},{"value":"Not applicable.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical Approval and Consent to Participate"}},{"value":"Not applicable.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for Publication"}}]}}