Superior human hepatocyte transduction with adeno-associated virus vector serotype 7
Open accessAbstract
Although therapeutic outcomes have been achieved in hemophilia patients after delivery of clotting factor genes to the liver using adeno-associated virus (AAV) vectors, it is well known that the preclinical results generated from hemophilia animal models have not been directly predictive of successful translation in humans. To address this discrepancy humanized mouse models have recently been used to predict AAV transduction efficiency for human hepatocytes. In this study we evaluated AAV vector transduction from several serotypes in human liver hepatocytes xenografted into chimeric mice. After systemic administration of AAV vectors encoding a GFP transgene in humanized mice, the liver was harvested for either immunohistochemistry staining or flow cytometry assay for AAV human hepatocyte transduction analysis. We observed that AAV7 consistently transduced human hepatocytes more efficiently than other serotypes in both immunohistochemistry assay and flow cytometry analysis. To better assess the future application of AAV7 for systemic administration in the treatment of hemophilia or other liver diseases, we analyzed the prevalence of neutralizing antibodies (NAbs) to AAV7 in sera from healthy subjects and patients with hemophilia. In the general population, the prevalence of NAbs to AAV7 was lower than that of AAV2 or AAV3B. However, a higher prevalence of AAV7 NAbs was found in patients with hemophilia. In summary, results from this study suggest that AAV7 vectors should be considered as an effective vehicle for human liver targeting in future clinical trials.
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AAV7 induces a stronger anti-AAV immune response in the hemophilia B population than AAV8, which makes AAV8 suitable for hemophilia B patients in clinical trials.
“Notably, AAV7 induces stronger anti-AAV immune response in the hemophilia B population than AAV8 [49], which makes AAV8 suitable for hemophilia B patients in clinical trials (Identifier: NCT01620801 and NCT00979238).”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [49]
The Results section (page unavailable) reports neutralizing antibodies to AAV7 in all 26 hemophilia patients tested, versus no detectable antibodies to AAV8 in six. But the study measured pre-existing antibodies, not whether AAV7 induces a stronger immune response, and it does not conclude that this comparison makes AAV8 suitable for clinical trials. (LLM, conf=0.87)
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- Authors
- Shao, W., Pei, X., Cui, C., Askew, C., Dobbins, A., Chen, X., Abajas, Y. L., Gerber, D. A., Samulski, R. J., Nichols, T. C., & Li, C.
- Affiliations
- University of North Carolina at Chapel Hill (US)
- Corresponding author
- Li, Chengwen
- ORCID
- Venue
- Gene Therapy
- Published
- 2019-09-30
- Volume
- 26
- Issue
- 12
- Pages
- 504-514
- ISSN
- 0969-7128 (ISSN-L)
- 1476-5462
- PMC
- PMC6923567
- OpenAlex
- W2976356593
- Cited by
- 22
- References
- 47
- Citations by year
- 2025: 3 · 2024: 5 · 2023: 3 · 2022: 3 · 2021: 3 · 2020: 5
- FWCI
- 1.66
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Gene Therapy (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- Funders
- U.S. Department of Health & Human Services | NIH | National Heart, Lung, and Blood Institute (P01HL112761, R01HL125749, R01HL144661)
- U.S. Department of Health & Human Services | NIH | National Institute of Allergy and Infectious Diseases (R01AI117408)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Medicine › Hematology › Hemophilia Treatment and Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- hepatocyte transduction, humanized mice, liver xenograft, hemophilia, neutralizing antibodies, AAV serotypes, systemic gene delivery, liver-directed gene therapy
- MeSH terms
- Dependovirus, Animals, Cell Line, Female, Genetic Vectors, Hemophilia A, Humans, Transduction, Genetic, Case-Control Studies, Hepatocytes, Green Fluorescent Proteins, Mice, Antibodies, Neutralizing, HEK293 Cells, Serogroup
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