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Updated: May 26, 2026
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Transgene Expression in Cultured Cells Using Unpurified Recombinant Adeno-Associated Viral Vectors
Published on: October 20, 2023
Self-complementary AAVs induce more potent transgene product-specific immune responses compared to a single-stranded
TeLang Wu1, Katrin Töpfer, Shih-Wen Lin
1The Wistar Institute, Philadelphia, Pennsylvania 19104, USA.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|December 22, 2011
Summary
Self-complementary adeno-associated virus (scAAV) vectors elicit stronger immune responses than single-stranded AAV (ssAAV) vectors. This enhanced immunogenicity may lead to adverse effects in gene therapy recipients due to increased transgene expression.
Area of Science:
- Gene therapy
- Immunology
- Virology
Background:
- Adeno-associated virus (AAV) vectors are widely used in gene therapy.
- Different AAV vector forms, single-stranded (ssAAV) and self-complementary (scAAV), have varying efficiencies.
- Immune responses to AAV vectors can impact gene transfer efficacy and safety.
Purpose of the Study:
- To compare the immunogenicity of scAAV and ssAAV vectors.
- To investigate the impact of AAV capsid serotypes on immune responses.
- To assess the relationship between transgene expression levels and immune reactions.
Main Methods:
- Utilized a mouse model for in vivo studies.
- Employed AAV vectors pseudotyped with serotypes 2, 7, and 8.
- Quantified transgene product-specific CD8(+) T cell and antibody responses.
Main Results:
- Self-complementary AAV vectors induced significantly more potent CD8(+) T cell and antibody responses compared to ssAAV vectors.
- The magnitude and speed of transgene product appearance correlated with immune response strength.
- Serotype 2, 7, and 8 capsids showed potent immune induction with scAAV vectors.
Conclusions:
- scAAV vectors demonstrate higher immunogenicity than ssAAV vectors.
- Increased and accelerated transgene expression from scAAV vectors may heighten detrimental immune responses.
- Findings have implications for designing safer and more effective gene transfer strategies.
