Rapid AAV-Neutralizing Antibody Determination with a Cell-Binding Assay
Open accessAbstract
Recombinant adeno-associated virus (rAAV) has been developed as a successful vector for both basic research and human gene therapy. However, neutralizing antibodies (NAbs) against AAV capsids can abolish AAV infectivity on target cells, reducing the transduction efficacy. Absence of AAV NAb has become a prerequisite qualification for patients enrolled in gene therapy trials. Nevertheless, accurate assessment of AAV NAb has remained a challenging task. Here we developed a rapid assay based on the observations that AAV NAb inhibits rAAV binding to the host cell surface and NAb titers are negatively related to the amount of AAV genomes binding to the target cells. By quantifying the AAV genome on the target cells in the presence of anti-sera, AAV NAb titers can be accurately determined. The titer determined by this assay correlates well with the classical transduction-based assays. A major advantage of this method is that it can be carried out with a 30-min binding assay without the lengthy wait for a transduction outcome. This assay is independent of transduction performance of AAV serotype in the target cells. Therefore, the AAV cell-binding assay for NAb determination offers an alternative method for in vivo NAb assay.
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About 50-90% of the human population have been exposed to different AAV serotypes.
“AAVs are widely spread in the natural environment, and it has been reported that about 50-90% of the human population have been exposed to different AAV serotypes [27].”
From Immune responses to gene editing by viral and non-viral delivery vectors used in retinal gene therapy (2022) · cites this paper as [27]
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Decoding Nonlinearities in AAV-Based Gene Therapy Using PBPK Modelling
Rajwade, A., Liu, S., Kumar, M., & Shah, D. K. (2026) · The AAPS Journal · doi:10.1208/s12248-026-01285-0
- Authors
- Guo, P., Zhang, J., Chrzanowski, M., Huang, J., Chew, H., Firrman, J. A., Sang, N., Diao, Y., & Xiao, W.
- Affiliations
- Huaqiao University (CN)
- Temple University (US)
- Drexel University (US)
- Corresponding author
- Diao, Yong
- Xiao, Weidong
- ORCID
- Venue
- Molecular Therapy - Methods & Clinical Development
- Publisher
- Elsevier BV
- Published
- 2019-06
- Volume
- 13
- Pages
- 40-46
- ISSN
- 2329-0501 (ISSN-L)
- PMC
- PMC6317305
- OpenAlex
- W2903251848
- Cited by
- 33
- References
- 23
- Citations by year
- 2026: 1 · 2025: 3 · 2024: 2 · 2023: 4 · 2022: 4 · 2021: 12 · 2020: 4 · 2019: 2 · 2017: 1
- FWCI
- 5.35
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Molecular Therapy — Methods & Clinical Development (publishedVersion, cc-by-nc-nd)
- Europe PMC (PubMed Central) (publishedVersion, cc-by-nc-nd)
- PubMed Central (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion)
- TUScholarShare (Temple University) (submittedVersion, cc-by-nc-nd)
- Funders
- National Institutes of Health (HL114152, HL130871)
- National Natural Science Foundation of China (81371669, 81371672)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Health Sciences › Medicine › Infectious Diseases › SARS-CoV-2 and COVID-19 Research
- Keywords
- adeno-associated virus, neutralizing antibodies, AAV capsid, gene therapy, preclinical screening, antibody titer measurement, transduction efficiency
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
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