ArticleGene therapy2015
Prediction of adeno-associated virus neutralizing antibody activity for clinical application.
Article in Gene therapy, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 32 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
32 citing papers in PubMed, 45 citations in OpenAlex.
- AAV2-mediated intravitreal delivery of exon-specific U1 snRNA rescues optic neuropathy in familial dysautonomia.Molecular therapy. Nucleic acids · 2026Article
- Neutralizing Antibodies: Role in Immune Response and Viral Vector Based Gene Therapy.International journal of molecular sciences · 2025Review
- CoreTIA: a modular, statistically robust transduction inhibition assay for AAV neutralization.Frontiers in immunology · 2025Article
- Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia.Gene therapy · 2024Article
- Understanding AAV vector immunogenicity: from particle to patient.Theranostics · 2024Review
- Structural and antigenic characterization of the avian adeno-associated virus capsid.Journal of virology · 2023Article
- Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Review
- Multiplexing AAV Serotype-Specific Neutralizing Antibodies in Preclinical Animal Models and Humans.Biomedicines · 2023Article
- Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials.Gene therapy · 2023Article
- Targeted Delivery for Cardiac Regeneration: Comparison of Intra-coronary Infusion and Intra-myocardial Injection in Porcine Hearts.Frontiers in cardiovascular medicine · 2022Article
- A sensitive and reproducible cell-based assay via secNanoLuc to detect neutralizing antibody against adeno-associated virus vector capsid.Molecular therapy. Methods & clinical development · 2021Article
- Anti-AAV Antibodies in AAV Gene Therapy: Current Challenges and Possible Solutions.Frontiers in immunology · 2021Review
- Adeno-Associated Viruses (AAV) and Host Immunity - A Race Between the Hare and the Hedgehog.Frontiers in immunology · 2021Review
- Modifiers of Adeno-Associated Virus-Mediated Gene Expression in Implication for Serotype-Universal Neutralizing Antibody Assay.Human gene therapy · 2020Article
- Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme.JCI insight · 2020Article
- Ocular Tolerability and Immune Response to Corneal Intrastromal AAV-Molecular therapy. Methods & clinical development · 2020Article
- Therapeutic hFIX Activity Achieved after Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs.Molecular therapy. Methods & clinical development · 2019Article
- Immunoadsorption enables successful rAAV5-mediated repeated hepatic gene delivery in nonhuman primates.Blood advances · 2019Article
- Rapid AAV-Neutralizing Antibody Determination with a Cell-Binding Assay.Molecular therapy. Methods & clinical development · 2019Article
- Threshold for Pre-existing Antibody Levels Limiting Transduction Efficiency of Systemic rAAV9 Gene Delivery: Relevance for Translation.Molecular therapy. Methods & clinical development · 2019Article
Corrections and comments
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Authors and funding
8 authors at 2 institutions in 2 countries.
Funding
Abstract
Patients with neutralizing antibodies (Nabs) against adeno-associated virus (AAV) are usually excluded from the treatment with AAV vectors. To develop a standard assay for detecting Nab inhibition activity, we systematically studied current AAV Nab assays in vitro and in vivo. Several factors were found that influence the Nab titers based on the in vitro assay, including sera volume, AAV dose per cell, cell number and choice of transgenes. When the Nab titer assay was performed in vivo via intramuscular (IM) or systemic administration, a fourfold increase in sensitivity for measurement of Nab titers was observed compared with an identical in vitro test. To better mimic the clinical setting, after passively transferring human Nabs into mice, blood was collected before systemic injection of AAV vector and used for Nab titer analysis in vitro or via IM injection. The results showed that AAV delivered via IM injection had a similar inhibition pattern to systemic administration. These studies indicate critical parameters necessary for optimizing Nab sensitivity and that an in vivo Nab assay is more sensitive than an in vitro assay for inclusion/exclusion criteria. The variables identified by this study may explain some of the compounding clinical data seen to date with respect to efficiency of AAV transduction in various phase I clinical trials.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.