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Updated: Aug 1, 2025
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Production, Purification, and Quality Control for Adeno-associated Virus-based Vectors
Published on: January 29, 2019
36.6K
Adeno-associated virus vector system controlling capsid expression improves viral quantity and quality.
Kenji Ohba1, Yoshihide Sehara1, Tatsuji Enoki2
1Division of Genetic Therapeutics, Center for Molecular Medicine, Jichi Medical University, Shimotsuke, Tochigi 329-0498, Japan.
Iscience
|April 25, 2023
Summary
This study developed a novel adeno-associated virus (AAV) production system. Timing capsid expression with tetracycline control significantly boosted viral yield and reduced empty capsids for gene therapy applications.
Area of Science:
- Gene Therapy
- Molecular Biology
- Virology
Background:
- Adeno-associated virus (AAV) vectors are crucial for gene therapy.
- Current AAV production generates numerous empty capsids, increasing costs.
- Efficient AAV production is vital for clinical gene therapy success.
Purpose of the Study:
- To develop an improved AAV production system.
- To regulate AAV capsid expression timing.
- To reduce empty capsid formation and enhance viral yield.
Main Methods:
- Utilized a tetracycline-dependent promoter to control capsid expression timing.
- Developed a novel AAV production system.
- Evaluated viral yield, empty capsid levels, and infectivity in vitro and in vivo across various serotypes.
Main Results:
- Tetracycline-regulated capsid expression increased overall viral yield.
- Reduced the proportion of empty capsids in multiple AAV serotypes.
- Maintained AAV vector infectivity in vitro and in vivo.
Conclusions:
- The developed AAV production system enhances viral quantity and quality.
- Timing capsid expression is an effective strategy to minimize empty capsids.
- This approach offers a new perspective for optimizing AAV vector production in gene therapy.
