Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6
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We present a simple and safe strategy for producing high-titer adeno-associated virus (AAV) vectors derived from six different AAV serotypes (AAV-1 to AAV-6). The method, referred to as "HOT," is helper virus free, optically controllable, and based on transfection of only two plasmids, i.e., an AAV vector construct and one of six novel AAV helper plasmids. The latter were engineered to carry AAV serotype rep and cap genes together with adenoviral helper functions, as well as unique fluorescent protein expression cassettes, allowing confirmation of successful transfection and identification of the transfected plasmid. Cross-packaging of vector DNA derived from AAV-2, -3, or -6 was up to 10-fold more efficient using our novel plasmids, compared to a conservative adenovirus-dependent method. We also identified a variety of useful antibodies, allowing detection of Rep or VP proteins, or assembled capsids, of all six AAV serotypes. Finally, we describe unique cell tropisms and kinetics of transgene expression for AAV serotype vectors in primary or transformed cells from four different species. In sum, the HOT strategy and the antibodies presented here, together with the reported findings, should facilitate and support the further development of AAV serotype vectors as powerful new tools for human gene therapy.
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For the majority of rAAV vectors, the ITRs and rep gene of AAV2 are utilized, resulting in pseudotyped rAAVs.
“For the majority of rAAV vectors, the ITRs and rep gene of serotype 2 (AAV2) are utilized resulting in pseudo-typed rAAVs [11].”
From Understanding Capsid Assembly and Genome Packaging for Adeno-Associated Viruses (2017) · cites this paper as [11]
The Results describe pseudotyped particles containing AAV-2 vector DNA made with a helper that retains AAV-2 rep while substituting capsid genes from other serotypes. The Discussion says pseudotyping of non-type-2 vectors had been described only once, supporting the prominence of AAV-2-based pseudotyping. Neither passage establishes that the majority of all rAAV vectors use AAV-2 ITRs and rep or that most rAAV vectors are pseudotyped. (LLM, conf=0.78)
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- Authors
- Grimm, D., Kay, M. A., & Kleinschmidt, J. A.
- Affiliations
- Stanford Medicine (US)
- German Cancer Research Center (DE)
- Corresponding author
- Kay, Mark A
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2003-06
- Volume
- 7
- Issue
- 6
- Pages
- 839-850
- ISSN
- 1525-0016 (ISSN-L)
- OpenAlex
- W1977797303
- Cited by
- 344
- References
- 36
- Citations by year
- 2026: 4 · 2025: 9 · 2024: 12 · 2023: 10 · 2022: 15 · 2021: 17 · 2020: 12 · 2019: 18 · 2018: 5 · 2017: 17 · 2016: 7 · 2015: 21 · 2014: 12 · 2013: 20 · 2012: 19
- FWCI
- 7.96
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Molecular Therapy (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- Funders
- National Heart, Lung, and Blood Institute (HL66948, U01HL066948)
- 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
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Viral Infectious Diseases and Gene Expression in Insects
- Keywords
- adeno-associated virus vectors, fluorescent protein markers, cell tropism, transgene expression kinetics, gene therapy vectors
- MeSH terms
- Dependovirus, Cell Line, DNA Replication, DNA-Binding Proteins, Genetic Vectors, Helper Viruses, Humans, Plasmids, Serotyping, Viral Proteins, Virus Replication, Virus Assembly
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