ArticleGene therapy2023
Large-scale purification of functional AAV particles packaging the full genome using short-term ultracentrifugation with a zonal rotor.
Article in Gene therapy, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
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Who cites it
22 citing papers in PubMed, 36 citations in OpenAlex.
- Automated zonal-rotor CsCl ultracentrifugation with ÄKTA-based fractionation removes empty and intermediate AAV capsids.Molecular therapy. Advances · 2026Article
- AAVR disruption as a producer-cell engineering strategy for enhanced supernatant-based recovery of selected AAV serotypes.Molecular therapy. Advances · 2026Article
- Removal of Empty AAV Capsids to Undetectable Levels Using Orthogonal Purification Steps.Biotechnology and bioengineering · 2026Article
- Gene modification: Exploring the potential in treating kidney diseases.Pharmacological research · 2026Review
- Lyophilization of Adeno-Associated Virus Serotypes for Storage and Global Distribution.Biomedicines · 2025Article
- AAV-PHP.eB achieves superior neuronal transduction over AAV9 in pigtail macaques following intracerebroventricular administration.Molecular therapy. Methods & clinical development · 2025Article
- Characterization of difficult-to-remove host cell proteins in adeno-associated virus downstream processing.Molecular therapy. Methods & clinical development · 2025Article
- Establishment of a novel human amniotic epithelial-derived cell line, HAT, for high-yield AAV vector production.Molecular therapy. Methods & clinical development · 2025Article
- AAV yield, bioactivity, and particle heterogeneity are impacted by genome size and non-coding DNA elements.Molecular therapy. Methods & clinical development · 2025Article
- AAV-Based Gene Therapy: Opportunities, Risks, and Scale-Up Strategies.International journal of molecular sciences · 2025Review
- Use of cesium chloride density gradient ultracentrifugation for the purification and characterization of recombinant adeno-associated virus.European biophysics journal : EBJ · 2025Article
- A two-pass anion-exchange chromatography strategy for enrichment of full capsids in manufacturing of adeno-associated viral vectors.Molecular therapy. Methods & clinical development · 2025Article
- Chromatography in Downstream Processing of Recombinant Adeno-Associated Viruses: A Review of Current and Future Practises.Biotechnology and bioengineering · 2025Review
- Identification of the role of SNARE proteins in rAAV vector production through interaction with the viral MAAP.Molecular therapy. Methods & clinical development · 2025Article
- Article
- Membrane Filtration of Nanoscale Biomaterials: Model System and Membrane Performance Evaluation for AAV2 Viral Vector Clarification and Recovery.Nanomaterials (Basel, Switzerland) · 2025Article
- Precision genome editing using combinatorial viral vector delivery of CRISPR-Cas9 nucleases and donor DNA constructs.Nucleic acids research · 2025Article
- A Novel Method for Separating Full and Empty Adeno-Associated Viral Capsids Using Ultrafiltration.Membranes · 2024Article
- Therapeutic Application and Structural Features of Adeno-Associated Virus Vector.Current issues in molecular biology · 2024Review
- In-Depth Comparison of Adeno-Associated Virus Containing Fractions after CsCl Ultracentrifugation Gradient Separation.Viruses · 2024Article
Corrections and comments
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Authors and funding
7 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Adeno-associated virus (AAV) vector-based gene therapy is potentially curative for various genetic diseases; however, the development of a scalable purification method for full-genome AAV vectors remains crucial to increase productivity and reduce cost of GMP production. In this study, we developed a large-scale short-term purification method for functional full-genome AAV particles by using 2-step cesium chloride (CsCl) density-gradient ultracentrifugation with a zonal rotor. The 2-step CsCl method with a zonal rotor improves separation between empty and full-genome AAV particles, reducing the ultracentrifugation time (4-5 h) and increasing the AAV volume for purification. The highly purified full-genome AAV particles were confirmed by analytical ultracentrifugation (AUC), droplet digital PCR (ddPCR) in the whole region of the AAV vector genome, transduction efficiency in target cells, and transmission electronic microscopy (TEM). The high-purity AAV9 particles were obtained using culture supernatant during vector preparation rather than cell lysate. CsCl could be simply removed by a hydroxyapatite column. Interestingly, ddPCR analysis revealed that "empty" AAV particles contain small fragments of the inverted terminal repeat (ITR), probably due to unexpected packaging of Rep-mediated ITR fragments. This large-scale functional AAV vector purification with ultracentrifugation would be effective for gene therapy.
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