AAVrh.10 immunogenicity in mice and humans. Relevance of antibody cross-reactivity in human gene therapy

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

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Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield

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Immune responses to AAV vectors: overcoming barriers to successful gene therapy

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Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses

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Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons

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Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

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Pre-existing Anti–Adeno-Associated Virus Antibodies as a Challenge in AAV Gene Therapy

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Adeno-Associated Virus Antibody Profiles in Newborns, Children, and Adolescents

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Adeno-associated viruses undergo substantial evolution in primates during natural infections

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Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates

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Intracerebral Administration of Adeno-Associated Viral Vector Serotype rh.10 Carrying Human <i>SGSH</i> and <i>SUMF1</i> cDNAs in Children with Mucopolysaccharidosis Type IIIA Disease: Results of a Phase I/II Trial

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Humoral immune response to AAV

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Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxia

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Adeno-Associated Virus Vectorology, Manufacturing, and Clinical Applications

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Long-term expression and repeated administration of AAV type 1, 2 and 5 vectors in skeletal muscle of immunocompetent adult mice

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Enhanced Survival of the LINCL Mouse Following CLN2 Gene Transfer Using the rh.10 Rhesus Macaque-derived Adeno-associated Virus Vector

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AAV9-mediated Expression of a Non-self Protein in Nonhuman Primate Central Nervous System Triggers Widespread Neuroinflammation Driven by Antigen-presenting Cell Transduction

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The Pleiotropic Effects of Natural AAV Infections on Liver-directed Gene Transfer in Macaques

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Capsid antibodies to different adeno-associated virus serotypes bind common regions

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Enhancing the Clinical Potential of AAV Vectors by Capsid Engineering to Evade Pre-Existing Immunity

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Cerebral infusion of AAV9 vector-encoding non-self proteins can elicit cell-mediated immune responses

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Adeno-Associated Virus Capsid Structure Drives CD4-Dependent CD8+ T Cell Response to Vector Encoded Proteins

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Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis

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Correction of Brain Oligodendrocytes by AAVrh.10 Intracerebral Gene Therapy in Metachromatic Leukodystrophy Mice

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Schwann cell targeting via intrasciatic injection of AAV8 as gene therapy strategy for peripheral nerve regeneration

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Intrapleural Administration of an AAVrh.10 Vector Coding for Human α1-Antitrypsin for the Treatment of α1-Antitrypsin Deficiency

Chiuchiolo, M. J., Kaminsky, S. M., Sondhi, D., Hackett, N. R., Rosenberg, J. B., Frenk, E. Z., Hwang, Y., de Graaf, B. G. V., Hutt, J. A., Wang, G., et al. (2013) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2013.168 · Cited by 52 · see citing works

Production of Recombinant Adeno-Associated Virus Vectors

Zolotukhin, S. (2005) · Human Gene Therapy · doi:10.1089/hum.2005.16.551 · Cited by 50 · see citing works

GENETIC FACTORS CONTROLLING ANTI-SHEEP ERYTHROCYTE ANTIBODY RESPONSE AND IMMUNOGLOBULIN SYNTHESIS IN BACKCROSS AND F2 PROGENY OF MICE GENETICALLY SELECTED FOR "HIGH" OR "LOW" ANTIBODY SYNTHESIS

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Intrathecal administration of IGF-I by AAVrh10 improves sensory and motor deficits in a mouse model of diabetic neuropathy

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Gene transfer to the CNS is efficacious in immune-primed mice harboring physiologically relevant titers of anti-AAV antibodies

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Characterization of Naturally-Occurring Humoral Immunity to AAV in Sheep

Tellez, J., Van Vliet, K., Tseng, Y.-S., Finn, J. D., Tschernia, N., Almeida-Porada, G., Arruda, V. R., Agbandje-McKenna, M., & Porada, C. D. (2013) · PLoS ONE · doi:10.1371/journal.pone.0075142 · Cited by 18 · see citing works

AAV vectors: The Rubik’s cube of human gene therapy

Pupo, A., Fernández, A., Low, S. H., François, A., Suárez-Amarán, L., & Samulski, R. J. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.09.015 · Cited by 361 · see citing works

Gene therapy advances: A meta-analysis of AAV usage in clinical settings

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Unraveling the complex story of immune responses to AAV vectors trial after trial.

Vandamme, C., Adjali, O., & Mingozzi, F. (2017) · Human Gene Therapy · doi:10.1089/hum.2017.150 · Cited by 234 · see citing works

Multifunctional CRISPR-Cas9 with Engineered Immunosilenced Human T Cell Epitopes

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AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease

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T-Cell Dependent Immunogenicity of Protein Therapeutics Pre-clinical Assessment and Mitigation–Updated Consensus and Review 2020

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Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations

Mendell, J. R., Connolly, A. M., Lehman, K. J., Griffin, D. A., Khan, S. Z., Dharia, S. D., Quintana-Gallardo, L., & Rodino-Klapac, L. R. (2022) · Molecular Therapy - Methods &amp; Clinical Development · doi:10.1016/j.omtm.2022.02.011 · Cited by 124 · see citing works

Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1 G93A Mice and Nonhuman Primates

Borel, F., Gernoux, G., Cardozo, B. H., Metterville, J., Cabrera, G. T., Song, L., Su, Q., Gao, G. P., ElMallah, M. K., Brown, R. H., et al. (2015) · Human Gene Therapy · doi:10.1089/hum.2015.122 · Cited by 111 · see citing works

Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects

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Identification of liver-specific enhancer–promoter activity in the 3′ untranslated region of the wild-type AAV2 genome

Logan, G. J., Dane, A. P., Hallwirth, C. V., Smyth, C. M., Wilkie, E. E., Amaya, A. K., Zhu, E., Khandekar, N., Ginn, S. L., Liao, S. H. Y., et al. (2017) · Nature Genetics · doi:10.1038/ng.3893 · Cited by 105 · see citing works

Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice

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AAV ablates neurogenesis in the adult murine hippocampus

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Evading and overcoming AAV neutralization in gene therapy

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AAV natural infection induces broad cross-neutralizing antibody responses to multiple AAV serotypes in chimpanzees

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Adeno-associated Vector Toxicity—To Be or Not to Be?

Büning, H., & Schmidt, M. (2015) · Molecular Therapy · doi:10.1038/mt.2015.182 · Cited by 71 · see citing works

Site-specific PEGylated adeno-associated viruses with increased serum stability and reduced immunogenicity

Yao, T., Zhou, X., Zhang, C., Yu, X., Tian, Z., Zhang, L., & Zhou, D. (2017) · Molecules · doi:10.3390/molecules22071155 · Cited by 68 · see citing works

Comparative Analysis of the Capsid Structures of AAVrh.10, AAVrh.39, and AAV8

Mietzsch, M., Barnes, C., Hull, J. A., Chipman, P. R., Xie, J., Bhattacharya, N., Sousa, D. R., McKENNA, R. V., Gao, G., & Agbandje‐McKenna, M. (2019) · Journal of Virology · doi:10.1128/jvi.01769-19 · Cited by 66 · see citing works

Strategies to circumvent humoral immunity to adeno-associated viral vectors

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Recommendations for the Development of Cell-Based Anti-Viral Vector Neutralizing Antibody Assays

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Humoral immune responses to <scp>AAV</scp> gene therapy in the ocular compartment

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Recent advances in gene therapy for lysosomal storage disorders

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AAV capsid engineering identified two novel variants with improved in vivo tropism for cardiomyocytes

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Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec

Day, J. W., Finkel, R. S., Mercuri, E., Swoboda, K. J., Menier, M., van Olden, R., Tauscher-Wisniewski, S., & Mendell, J. R. (2021) · Molecular Therapy - Methods &amp; Clinical Development · doi:10.1016/j.omtm.2021.02.014 · Cited by 48 · see citing works

Cryo-electron Microscopy of Adeno-associated Virus

Stagg, S. M., Yoshioka, C., Davulcu, O., & Chapman, M. S. (2022) · Chemical Reviews · doi:10.1021/acs.chemrev.1c00936 · Cited by 47 · see citing works

Delivering gene therapy for mucopolysaccharide diseases

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Advances in the treatment of neuronal ceroid lipofuscinosis

Rosenberg, J. B., Chen, A., Kaminsky, S. M., Crystal, R. G., & Sondhi, D. (2019) · Expert Opinion on Orphan Drugs · doi:10.1080/21678707.2019.1684258 · Cited by 41 · see citing works

Liver‐directed gene therapy for inherited metabolic diseases

Baruteau, J., Brunetti‐Pierri, N., & Gissen, P. (2024) · Journal of Inherited Metabolic Disease · doi:10.1002/jimd.12709 · Cited by 45 · see citing works

An AAVrh10-CAG-CYP21-HA vector allows persistent correction of 21-hydroxylase deficiency in a Cyp21−/− mouse model

Perdomini, M., Dos Santos, C., Goumeaux, C., Blouin, V, & Bougnères, P. (2017) · Gene Therapy · doi:10.1038/gt.2017.10 · Cited by 37 · see citing works

Gene therapy for hemophilia – From basic science to first approvals of “one-and-done” therapies

Herzog, R. W., Kaczmarek, R., & High, K. A. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.03.043 · Cited by 37 · see citing works

Comparison of Serum rAAV Serotype-Specific Antibodies in Patients with Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Inclusion Body Myositis, or GNE Myopathy

Zygmunt, D. A., Crowe, K. E., Flanigan, K. M., & Martin, P. T. (2017) · Human Gene Therapy · doi:10.1089/hum.2016.141 · Cited by 36 · see citing works

Recombinant Adeno-Associated Virus Vectors for Gene Therapy of the Central Nervous System: Delivery Routes and Clinical Aspects

Słyk, Ż., Stachowiak, N., & Małecki, M. (2024) · Biomedicines · doi:10.3390/biomedicines12071523 · Cited by 39 · see citing works

Efficacy of AAV serotypes to target Schwann cells after intrathecal and intravenous delivery

Kagiava, A., Richter, J., Tryfonos, C., Leal-Julià, M., Sargiannidou, I., Christodoulou, C., Bosch, A., & Kleopa, K. A. (2021) · Scientific Reports · doi:10.1038/s41598-021-02694-1 · Cited by 34 · see citing works

Megalencephalic Leukoencephalopathy: Insights Into Pathophysiology and Perspectives for Therapy

Bosch, A., & Estévez, R. (2021) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2020.627887 · Cited by 28 · see citing works

Development of a Chemiluminescent ELISA Method for the Detection of Total Anti-Adeno Associated Virus Serotype 9 (AAV9) Antibodies

Kavita, U., Dai, Y., Salvador, L., Miller, W., Adam, L. P., Lévesque, P., Zhang, Y. J., Ji, Q., & Pillutla, R. C. (2018) · Human Gene Therapy Methods · doi:10.1089/hgtb.2018.131 · Cited by 23 · see citing works

Induction of T-Cell Infiltration and Programmed Death Ligand 2 Expression by Adeno-Associated Virus in Rhesus Macaque Skeletal Muscle and Modulation by Prednisone

Cramer, M. L., Shao, G., Rodino-Klapac, L. R., Chicoine, L. G., & Martin, P. T. (2017) · Human Gene Therapy · doi:10.1089/hum.2016.113 · Cited by 22 · see citing works

Recombinant adeno-associated virus vectors in the treatment of rare diseases

Hastie, E. L., & Samulski, R. J. (2015) · Expert Opinion on Orphan Drugs · doi:10.1517/21678707.2015.1039511 · Cited by 22 · see citing works

Changing Fate: Reprogramming Cells via Engineered Nanoscale Delivery Materials

Dehnavi, S. S., Zadeh, Z. E., Harvey, A. R., Voelcker, N. H., Parish, C. L., Williams, R. J., Elnathan, R., & Nisbet, D. R. (2022) · Advanced Materials · doi:10.1002/adma.202108757 · Cited by 22 · see citing works

Anti-Phospho-Tau Gene Therapy for Chronic Traumatic Encephalopathy

Sacramento, C. B., Sondhi, D., Rosenberg, J. B., Chen, A., Giordano, S., Pey, E. P., Lee, V., Stiles, K. M., Havlicek, D. F., Leopold, P. L., et al. (2019) · Human Gene Therapy · doi:10.1089/hum.2019.174 · Cited by 21 · see citing works

Optimized AAV rh. 10 vectors that partially evade neutralizing antibodies during hepatic gene transfer

Selot, R., Arumugam, S., Mary, B., Cheemadan, S., & Jayandharan, G. R. (2017) · Frontiers in Pharmacology · doi:10.3389/fphar.2017.00441 · Cited by 20 · see citing works

Inner Ear Gene Therapy: An Overview from Bench to Bedside

Gadenstaetter, A. J., Krumpoeck, P. E., & Landegger, L. D. (2024) · Molecular Diagnosis & Therapy · doi:10.1007/s40291-024-00759-1 · Cited by 23 · see citing works

Prevalence of Adeno-Associated Virus 3 Capsid Binding and Neutralizing Antibodies in Healthy and Hemophilia B Individuals from India

Daniel, H. D. J., Kumar, S., Kannangai, R., Lakshmi, K. M., Agbandje‐McKenna, M., Coleman, K. E., Srivastava, A., Srivastava, A., & Abraham, A. M. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.258 · Cited by 19 · see citing works

Structural Study of Aavrh.10 Receptor and Antibody Interactions

Mietzsch, M., Yu, J. C., Hsi, J., Chipman, P. R., Broecker, F., Fuming, Z., Linhardt, R. J., Seeberger, P. H., Heilbronn, R., McKenna, R., et al. (2021) · Journal of Virology · doi:10.1128/jvi.01249-21 · Cited by 16 · see citing works

Preclinical studies in Krabbe disease: A model for the investigation of novel combination therapies for lysosomal storage diseases

Heller, G., Bradbury, A. M., Sands, M. S., & Bongarzone, E. R. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.09.017 · Cited by 17 · see citing works

High-Density Recombinant Adeno-Associated Viral Particles are Competent Vectors for In Vivo Transduction

Qizhao, W., Firrman, J., Wu, Z., Pokiniewski, K. A., Valencia, C. A., Wang, H., Wei, H., Zhuang, Z., Liu, L., Wunder, S. L., et al. (2016) · Human Gene Therapy · doi:10.1089/hum.2016.055 · Cited by 17 · see citing works

Low Seroprevalence of Neutralizing Antibodies Targeting Two Clade F AAV in Humans

Ellsworth, J. L., O'Callaghan, M., Rubin, H. L., & Seymour, A. B. (2018) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2017.239 · Cited by 14 · see citing works

AAVrh-10 transduces outer retinal cells in rodents and rabbits following intravitreal administration

Zeng, Y., Qian, H., Wu, Z., Marangoni, D., Sieving, P. A., & Bush, R. A. (2019) · Gene Therapy · doi:10.1038/s41434-019-0094-3 · Cited by 12 · see citing works

Prevalence Study of Cellular Capsid-Specific Immune Responses to AAV2, 4, 5, 8, 9, and rh10 in Healthy Donors

Xicluna, R., Avenel, A., Vandamme, C., Devaux, M., Jaulin, N., Couzinié, C., Duff, J. L., Charrier, A., Guilbaud, M., Adjali, O., et al. (2024) · Human Gene Therapy · doi:10.1089/hum.2023.225 · Cited by 12 · see citing works

Intracerebral lentiviral ABCD1 gene therapy in an early disease onset ALD mouse model

Gong, J., Liu, Y., Chung, T., Xu, L., Lund, T. C., & Chang, L. (2022) · Gene Therapy · doi:10.1038/s41434-022-00355-0 · Cited by 12 · see citing works

Cerebellar Astrocyte Transduction as Gene Therapy for Megalencephalic Leukoencephalopathy

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Gene therapy for leukodystrophy: progress, challenges and opportunities

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