Production of adeno-associated virus vectors for in vitro and in vivo applications
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Delivering and expressing a gene of interest in cells or living animals has become a pivotal technique in biomedical research and gene therapy. Among viral delivery systems, adeno-associated viruses (AAVs) are relatively safe and demonstrate high gene transfer efficiency, low immunogenicity, stable long-term expression, and selective tissue tropism. Combined with modern gene technologies, such as cell-specific promoters, the Cre/lox system, and genome editing, AAVs represent a practical, rapid, and economical alternative to conditional knockout and transgenic mouse models. However, major obstacles remain for widespread AAV utilization, such as impractical purification strategies and low viral quantities. Here, we report an improved protocol to produce serotype-independent purified AAVs economically. Using a helper-free AAV system, we purified AAVs from HEK293T cell lysates and medium by polyethylene glycol precipitation with subsequent aqueous two-phase partitioning. Furthermore, we then implemented an iodixanol gradient purification, which resulted in preparations with purities adequate for in vivo use. Of note, we achieved titers of 10 10 –10 11 viral genome copies per µl with a typical production volume of up to 1 ml while requiring five times less than the usual number of HEK293T cells used in standard protocols. For proof of concept, we verified in vivo transduction via Western blot, qPCR, luminescence, and immunohistochemistry. AAVs coding for glutaredoxin-1 (Glrx) shRNA successfully inhibited Glrx expression by ~66% in the liver and skeletal muscle. Our study provides an improved protocol for a more economical and efficient purified AAV preparation.
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Virus concentration in rAAV samples covers many orders of magnitude, from 10^10 to 10^11 vg/mL at raw culture harvest to 10^13 to 10^14 vg/mL in the purified product.
“At the same time, virus concentration in samples covers many orders of magnitude, from 1010 to 1011 vg/mL at raw culture harvest to 1013 to 1014 vg/mL in the purified product,6,7,18, 19, 20,24, 25, 26, 27”
From Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies (2021) · cites this paper as [6]
Laboratory-scale production yields only a few milliliters of purified rAAV, equivalent to 10^12 to 10^14 vector genomes, such that the required sample volumes for analysis can cause significant loss of product.
“Commonly, laboratory-scale production yields only few milliliters (mL) of purified rAAV—equivalent to 1012–1014 vector genomes (vgs)—such that the required sample volumes for analysis can cause significant loss of product.6,7,20,23,24”
From Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies (2021) · cites this paper as [6]
The ratio of full capsids to all capsids, the content ratio, can vary from less than 1% to 30% in the production culture at the time of harvest and is inconsistent between production runs.
“The ratio of full capsids to all capsids, termed content ratio, can vary from <1% to 30% in the production culture at the time of harvest and is inconsistent between production runs (Figure 1).6,28, 29, 30”
From Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies (2021) · cites this paper as [6]
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- Authors
- Kimura, T., Ferran, B., Tsukahara, Y., Shang, Q., Desai, S., Fedoce, A., Pimentel, D. R., Luptak, I., Adachi, T., Ido, Y., Matsui, R., & Bachschmid, M. M.
- Affiliations
- Boston University (US)
- National Defense Medical College Hospital (JP)
- Corresponding author
- Matsui, Reiko
- Bachschmid, Markus Michael
- ORCID
- Venue
- Scientific Reports
- Published
- 2019-09-19
- Volume
- 9
- Issue
- 1
- Pages
- 13601-13601
- ISSN
- 2045-2322 (ISSN-L)
- PMC
- PMC6753157
- OpenAlex
- W2973390883
- Cited by
- 155
- References
- 70
- Citations by year
- 2026: 18 · 2025: 30 · 2024: 27 · 2023: 27 · 2022: 23 · 2021: 20 · 2020: 10
- FWCI
- 7.98
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Locations
- Scientific Reports (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion)
- Funders
- American Heart Association (15FTF25890062, 16GRNT27660006)
- School of Medicine, Boston University (1UL1TR001430)
- National Defense Medical College
- Ministry of Education, Culture, Sports, Science and Technology
- Ministry of Defense
- National Institutes of Health (NIH T32, 15FTF25890062, UL1TR001430)
- Japan Society for the Promotion of Science (18H02815)
- National Institute on Aging (R03AG051857)
- National Heart, Lung, and Blood Institute (T32HL007224, R01HL133013)
- National Institute of Diabetes and Digestive and Kidney Diseases (R01DK103750)
- National Center for Advancing Translational Sciences
- 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, AAV production, HEK293T cells, polyethylene glycol precipitation, aqueous two-phase extraction, viral titer, in vivo transduction, glutaredoxin 1, shRNA, liver, skeletal muscle, gene therapy
- MeSH terms
- Proof of Concept Study, Dependovirus, Animals, Cell Line, Genetic Vectors, Humans, Liver, Male, Polyethylenes, Chemical Precipitation, Transduction, Genetic, Down-Regulation, Muscle, Skeletal, Viral Load, RNA, Small Interfering, Glutaredoxins, HEK293 Cells
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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