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Strategies to circumvent humoral immunity to adeno-associated viral vectors

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Yuhang Tian, Zhao Liu, Haoyan Tan, Jiahui Hou, Xin Wen, Fan Yang & Wen Cheng. (2020) New Aspects of Ultrasound-Mediated Targeted Delivery and Therapy for Cancer. International Journal of Nanomedicine 15, pages 401-418.
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Michael Hudecek, Zsuzsanna Izsvák, Sandra Johnen, Matthias Renner, Gabriele Thumann & Zoltán Ivics. (2017) Going non-viral: the Sleeping Beauty transposon system breaks on through to the clinical side. Critical Reviews in Biochemistry and Molecular Biology 52:4, pages 355-380.
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Bijay Dhungel, Aparna Jayachandran, Christopher J. Layton & Jason C. Steel. (2017) Seek and destroy: targeted adeno-associated viruses for gene delivery to hepatocellular carcinoma. Drug Delivery 24:1, pages 289-299.
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Sem J. Aronson, Ulrich Beuers & Piter J. Bosma. (2015) Progress and challenges in gene therapy for Crigler–Najjar syndrome. Expert Opinion on Orphan Drugs 3:12, pages 1387-1396.
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Articles from other publishers (42)

Sundeep Chandra, Brian R. Long, Carlos Fonck, Andrew C. Melton, Jeremy Arens, Jill Woloszynek & Charles A. O’Neill. (2023) Safety Findings of Dosing Gene Therapy Vectors in NHP With Pre-existing or Treatment-Emergent Anti-capsid Antibodies. Toxicologic Pathology.
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Andrea Vannini, Federico Parenti, Catia Barboni, Cristina Forghieri, Valerio Leoni, Mara Sanapo, Daniela Bressanin, Anna Zaghini, Gabriella Campadelli-Fiume & Tatiana Gianni. (2023) Efficacy of Systemically Administered Retargeted Oncolytic Herpes Simplex Viruses—Clearance and Biodistribution in Naïve and HSV-Preimmune Mice. Cancers 15:16, pages 4042.
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Maura K. Schwartz, Shibi Likhite, Tatyana A. Vetter, Megan C. Baird, Vicki McGovern, Andrea Sierra Delgado, Tom Mendel, Arthur Burghes & Kathrin C. Meyer. (2023) In-Depth Comparison of Anc80L65 and AAV9 Retinal Targeting and Characterization of Cross-Reactivity to Multiple AAV Serotypes in Humans. Molecular Therapy - Methods & Clinical Development.
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Motahareh Arjomandnejad, Ishani Dasgupta, Terence R. Flotte & Allison M. Keeler. (2023) Immunogenicity of Recombinant Adeno-Associated Virus (AAV) Vectors for Gene Transfer. BioDrugs 37:3, pages 311-329.
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Martin Schulz, Daniel I. Levy, Christos J. Petropoulos, George Bashirians, Ian Winburn, Matthias Mahn, Suryanarayan Somanathan, Seng H. Cheng & Barry J. Byrne. (2023) Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy. Molecular Therapy 31:3, pages 616-630.
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Lin Kang, Shilin Jin, Jiayi Wang, Zhongyue Lv, Chengqi Xin, Chengcheng Tan, Mengke Zhao, Liang Wang & Jing Liu. (2023) AAV vectors applied to the treatment of CNS disorders: Clinical status and challenges. Journal of Controlled Release 355, pages 458-473.
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Kai Wang, Min Zheng, Charles Askew, Xintao Zhang, Chengwen Li & Zongchao Han. (2022) Elastin‐Like Polypeptides Facilitate Adeno‐Associated Virus Transduction in the Presence of Pre‐Existing Neutralizing Antibodies. Advanced Therapeutics 5:11, pages 2200128.
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Rawa Arif, Klaus Kallenbach, Oliver J. Müller & Andreas H. Wagner. (2022) Gentherapie der TransplantatvaskulopathieGene therapy for transplant vasculopathy. Zeitschrift für Herz-,Thorax- und Gefäßchirurgie 36:5, pages 328-333.
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Scott M. Stagg, Craig Yoshioka, Omar Davulcu & Michael S. Chapman. (2022) Cryo-electron Microscopy of Adeno-associated Virus. Chemical Reviews 122:17, pages 14018-14054.
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Motahareh Arjomandnejad & Allison M. Keeler. (2022) Evaluating Readministration of Adeno-Associated Virus for Gene Therapy. Human Gene Therapy 33:5-6, pages 218-220.
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Alexander A. Iyer, Dimah Saade, Diana Bharucha-Goebel, A. Reghan Foley, Gilberto ‘Mike’ Averion, Eduardo Paredes, Steven Gray, Carsten G. Bönnemann, Christine Grady, Saskia Hendriks & Annette Rid. (2021) Ethical challenges for a new generation of early-phase pediatric gene therapy trials. Genetics in Medicine 23:11, pages 2057-2066.
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Amina Abulimiti, Michael Siu-Lun Lai & Raymond Chuen-Chung Chang. (2021) Applications of adeno-associated virus vector-mediated gene delivery for neurodegenerative diseases and psychiatric diseases: Progress, advances, and challenges. Mechanisms of Ageing and Development 199, pages 111549.
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Jenna Klotz, Carolina Tesi Rocha, Sally Dunaway Young, Tina Duong, MyMy Buu, Jacinda Sampson & John W. Day. (2021) Advances in the Therapy of Spinal Muscular Atrophy. The Journal of Pediatrics 236, pages 13-20.e1.
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Michael Whitehead, Andrew OsbornePatrick Yu‐Wai‐ManKeith Martin. (2021) Humoral immune responses to AAV gene therapy in the ocular compartment . Biological Reviews 96:4, pages 1616-1644.
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Natalie J. Holl, Han-Jung Lee & Yue-Wern Huang. (2021) Evolutionary Timeline of Genetic Delivery and Gene Therapy. Current Gene Therapy 21:2, pages 89-111.
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Lan Huang, Jie Wan, Yinqiu Wu, Yu Tian, Yizheng Yao, Shun Yao, Xiaoyun Ji, Shengjun Wang, Zhaoliang Su & Huaxi Xu. (2021) Challenges in adeno-associated virus-based treatment of central nervous system diseases through systemic injection. Life Sciences 270, pages 119142.
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Petr O. Ilyinskii, Alicia M. Michaud, Christopher J. Roy, Gina L. RizzoStephanie L. Elkins, Teresa Capela, Aparajita C. ChowdhurySheldon S. Leung & Takashi K. Kishimoto. (2021) Enhancement of liver-directed transgene expression at initial and repeat doses of AAV vectors admixed with ImmTOR nanoparticles. Science Advances 7:9.
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Anthony A. Stephenson & Kevin M. Flanigan. 2021. Curing Genetic Diseases Through Genome Reprogramming. Curing Genetic Diseases Through Genome Reprogramming 225 255 .
Zachary C. Elmore, Daniel K. Oh, Katherine E. Simon, Marco M. Fanous & Aravind Asokan. (2020) Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme. JCI Insight 5:19.
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L. Patrick Havlik, Katherine E. Simon, J. Kennon Smith, Kelli A. Klinc, Longping V. Tse, Daniel K. Oh, Marco M. Fanous, Rita M. Meganck, Mario Mietzsch, Jürgen Kleinschmidt, Mavis Agbandje-McKenna & Aravind Asokan. (2020) Coevolution of Adeno-associated Virus Capsid Antigenicity and Tropism through a Structure-Guided Approach. Journal of Virology 94:19.
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Nicola Salvatore Orefice. (2020) Development of New Strategies Using Extracellular Vesicles Loaded with Exogenous Nucleic Acid. Pharmaceutics 12:8, pages 705.
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Yi Shen, Robert E. Campbell, Daniel C. Côté & Marie-Eve Paquet. (2020) Challenges for Therapeutic Applications of Opsin-Based Optogenetic Tools in Humans. Frontiers in Neural Circuits 14.
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Longping V. Tse, Rita M. Meganck, Rachel L. Graham & Ralph S. Baric. (2020) The Current and Future State of Vaccines, Antivirals and Gene Therapies Against Emerging Coronaviruses. Frontiers in Microbiology 11.
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Natalie F. Nidetz, Michael C. McGee, Longping V. Tse, Chengwen Li, Le Cong, Yunxing Li & Weishan Huang. (2020) Adeno-associated viral vector-mediated immune responses: Understanding barriers to gene delivery. Pharmacology & Therapeutics 207, pages 107453.
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Anca Remes, Maximilian Franz, Franziska Mohr, Antje Weber, Kleopatra Rapti, Andreas Jungmann, Matthias Karck, Markus Hecker, Klaus Kallenbach, Oliver J. Müller, Rawa Arif & Andreas H. Wagner. (2019) AAV-Mediated Expression of AP-1-Neutralizing RNA Decoy Oligonucleotides Attenuates Transplant Vasculopathy in Mouse Aortic Allografts. Molecular Therapy - Methods & Clinical Development 15, pages 246-256.
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Nerea Zabaleta, Mirja Hommel, David Salas & Gloria Gonzalez-Aseguinolaza. (2019) Genetic-Based Approaches to Inherited Metabolic Liver Diseases. Human Gene Therapy 30:10, pages 1190-1203.
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Sylvie Da Rocha, Jérémy Bigot, Fanny Onodi, Jérémie Cosette, Guillaume Corre, Jérôme Poupiot, David Fenard, Bernard Gjata, Anne Galy & Thi My Anh Neildez-Nguyen. (2019) Temporary Reduction of Membrane CD4 with the Antioxidant MnTBAP Is Sufficient to Prevent Immune Responses Induced by Gene Transfer. Molecular Therapy - Methods & Clinical Development 14, pages 285-299.
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Fiona T. van den Berg, Nigel A. Makoah, Stuart A. Ali, Tristan A. Scott, Rutendo E. Mapengo, Lorraine Z. Mutsvunguma, Nonhlanhla N. Mkhize, Bronwen E. Lambson, Prudence D. Kgagudi, Carol Crowther, Salim S. Abdool Karim, Alejandro B. Balazs, Marc S. Weinberg, Abdullah Ely, Patrick B. Arbuthnot & Lynn Morris. (2019) AAV-Mediated Expression of Broadly Neutralizing and Vaccine-like Antibodies Targeting the HIV-1 Envelope V2 Region. Molecular Therapy - Methods & Clinical Development 14, pages 100-112.
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Ariana Jose, Mario Mietzsch, J. Kennon Smith, Justin Kurian, Paul Chipman, Robert McKenna, John Chiorini & Mavis Agbandje-McKenna. (2019) High-Resolution Structural Characterization of a New Adeno-associated Virus Serotype 5 Antibody Epitope toward Engineering Antibody-Resistant Recombinant Gene Delivery Vectors. Journal of Virology 93:1.
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Dongsheng Duan. (2018) Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy. Molecular Therapy 26:10, pages 2337-2356.
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Gerald Z. Zhuang, Udita Upadhyay, Xiaoying Tong, Yuan Kang, Diana M. Erasso, Eugene S. Fu, Konstantinos D. Sarantopoulos, Eden R. Martin, Tim Wiltshire, Luda Diatchenko, Shad B Smith, William Maixner & Roy C. Levitt. (2018) Human carbonic anhydrase-8 AAV8 gene therapy inhibits nerve growth factor signaling producing prolonged analgesia and anti-hyperalgesia in mice. Gene Therapy 25:4, pages 297-311.
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Xiaoxia Shi, Matthew Ryan Ykema, Jaco Hazenoot, Lysbeth ten Bloemendaal, Irene Mancini, Machteld Odijk, Peter de Haan & Piter J. Bosma. (2018) Cre Recombinase Mediates the Removal of Bacterial Backbone to Efficiently Generate rSV40. Molecular Therapy - Methods & Clinical Development 9, pages 225-233.
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J. Kennon Smith & Mavis Agbandje-McKenna. (2018) Creating an arsenal of Adeno-associated virus (AAV) gene delivery stealth vehicles. PLOS Pathogens 14:5, pages e1006929.
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Miguel G. Toscano, Jeroen van der Velden, Sybrand van der Werf, Machteld Odijk, Ana Roque, Rafael J. Camacho-Garcia, Irene G. Herrera-Gomez, Irene Mancini & Peter de Haan. (2017) Generation of a Vero-Based Packaging Cell Line to Produce SV40 Gene Delivery Vectors for Use in Clinical Gene Therapy Studies. Molecular Therapy - Methods & Clinical Development 6, pages 124-134.
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Celine VandammeOumeya ADJALIFederico Mingozzi. (2017) Unraveling the complex story of immune responses to AAV vectors trial after trial. Human Gene Therapy.
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Julien Baruteau, Simon N. Waddington, Ian E. Alexander & Paul Gissen. (2017) Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects. Journal of Inherited Metabolic Disease 40:4, pages 497-517.
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Longping Victor TseKelli A. KlincVictoria J. MadiganRuth M. Castellanos RiveraLindsey F. WellsL. Patrick HavlikJ. Kennon SmithMavis Agbandje-McKennaAravind Asokan. (2017) Structure-guided evolution of antigenically distinct adeno-associated virus variants for immune evasion. Proceedings of the National Academy of Sciences 114:24.
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A. N. Lukashev & A. A. ZamyatninJr.Jr.. (2016) Viral vectors for gene therapy: Current state and clinical perspectives. Biochemistry (Moscow) 81:7, pages 700-708.
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Michaël HocquemillerLaura GierschMickael AudrainSamantha ParkerNathalie Cartier. (2016) Adeno-Associated Virus-Based Gene Therapy for CNS Diseases. Human Gene Therapy 27:7, pages 478-496.
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