Self-Complementary AAV (scAAV) Particles
Product DetailsApplicationCase StudyFAQ
Product Details
Self-complementary adeno-associated virus (scAAV) features a dimeric inverted repeat genome, forming double-stranded DNA (dsDNA). scAAV exhibits several advantages over traditional single-stranded AAV, including enhanced and prolonged transgene expression both in vitro and in vivo. It can infect various cellular targets using different serotypes and offers higher in vivo DNA stability with more effective circularization. Creative Biogene provides prepackaged scAAV particles, saving time and costs. Contact us for custom services if you don't find the desired product.
Key Features of Our scAAV Particles
- Engineered Dimeric Genomes: Accelerate transgene expression, ensuring faster and higher yields.
- Rapid Transduction Kinetics: Achieve swift and robust gene delivery.
- Validation in Cells and Models: Proven efficacy in mammalian cells and animal studies.
- Custom scAAV Production: Tailored solutions for specific gene therapy needs.
Browse All scAAV Particles
Application
Creative Biogene scAAVs provide an enabling technology platform for overcoming critical bottlenecks. scAAV's dimeric genomes bypass second-strand synthesis requirements for transduction, overcoming rate-limiting steps in single-stranded AAV infection. This series of products features the following characteristics and applications:
- Neuronal and Glial Cell Transduction: Achieves efficient transduction of cells in the nervous system, including neurons and glial cells. Applied in the study of gene expression related to the nervous system.
- Rapid and Stable Transgene Expression: Provides rapid and stable transgene expression applicable to a wide range of cell lines and animal models. Utilized for gene function studies and drug screening.
- Mediation of Cre Recombinase Expression: Facilitates efficient mediation of Cre recombinase expression, suitable for genetic experiments and gene editing research.
- Highly Efficient Transduction of Specific Cell Types: Applicable for highly efficient transduction of specific cell types, useful for drug screening and gene function studies.
Case Study
Case Study 1
Researchers employed self-complementary adeno-associated virus (scAAV) to deliver Cas9, and single-stranded AAV (ssAAV) for delivering guide RNAs into DMD model mice. The application results of the dual AAV system demonstrated that researchers needed over 20 times lower doses of scAAV for efficient genome editing compared to ssAAV. The treated mice exhibited restored dystrophin expression and improved muscle function. Therefore, the utilization of scAAV for CRISPR-Cas9 delivery significantly enhances editing efficiency in DMD, marking a pivotal advancement toward clinical application.
Figure 1. scAAV delivery of sgRNAs achieved 20-70x higher editing efficiency than ssAAV in vitro, with 50% of INDELs restoring the dystrophin reading frame. (Zhang Y, et al., 2020).
Case Study 2
Researchers utilized adeno-associated virus (AAV) and generated single-chain antibodies against the TAR DNA-binding protein-43 (TDP-43) RRM1 domain, a hallmark of degenerating neurons in amyotrophic lateral sclerosis (ALS) and subsets of frontotemporal dementia (FTD). The delivery of the VH7Vk9 antibody via self-complementary AAV (scAAV) into mouse nervous systems resulted in reduced microgliosis, cognitive and motor defects, TDP-43 pathology, and inflammation in transgenic ALS-TDP-43 mice. This suggests that RRM1-targeted antibodies delivered by AAVs may offer new therapeutic approaches for ALS and FTD.
Figure 2. The researchers injected scAAV2/9 viruses encoding VH7Vk9 or 8H11 (anti-GFP) scFv antibodies into slices expressing a genomic segment encoding the human TDP-43 series G348C, a mouse model that exhibits TDP- Cytoplasmic accumulation of 43 in neurons. (Pozzi S, et al., 2019).
FAQ
A: scAAV is a self-complementary adeno-associated virus subtype with a genome containing dimeric inverted repeat sequences, allowing it to form double-stranded DNA structures. This structure contributes to increased efficiency and stability in gene expression.
Q: How does the double-stranded genome of scAAV impact DNA stability and circularization efficiency?
A: The double-stranded genome of scAAV, post-cell transduction, eliminates the need for a second-strand synthesis step, thereby enhancing DNA stability. Additionally, the double-stranded structure facilitates more efficient DNA circularization, improving the persistence of transgene expression.
A: scAAV is chosen for scenarios demanding rapid, efficient gene expression with high requirements for gene stability and circularization efficiency. Particularly in applications seeking prolonged-expression and enhanced transduction efficiency, scAAV proves to be an ideal selection.
Q: What advantages does scAAV have over traditional single-stranded AAV in terms of transduction kinetics and efficacy?
A: scAAV exhibits faster transduction kinetics compared to single-stranded AAV, as it bypasses the second-strand synthesis step. Its double-stranded genome structure enhances the efficiency and stability of gene expression, making it superior in quick and efficient gene delivery. Furthermore, scAAV outperforms DNA stability and circularization efficiency.
* For research use only. Not intended for any clinical use.