Adeno-associated virus (AAV)
Engineered AAV vectors for in-vivo gene expression and knockdown
In vivo expression and knockdown experiments help uncover the phenotypic impact of a genetic modification on the level of the living organism. Adeno-associated viruses (AAVs) are ideal for this kind of application.
Benefits of AAV:
- Lack of apparent pathogenicity → low immunogenicity
- Long term gene expression in vivo (> 1 year)
- Infects dividing and non-dividing cells
- Different serotypes/ promoters allow for highest tissue specificity
- BL-1 (S1) compatible in Germany (other safety regulations might apply in other countries)
SIRION BIOTECH masters FPLC-grade AAV production and engineering services, providing high quality products in less than 8 weeks. The sophisticated platform allows the integration of virtually any desired expression construct within the vector’s cloning capacity. Batch sizes range from 5×1011 GC (genetic copies) for small explorative experiments all the way to 5×1014 GC to satisfy complete experimental cohorts.
Special Feature – AAV OPTIMIZATION FOR GENE THERAPY
SIRION BIOTECH offers a range of several different AAV serotypes in combination with tissue specific promotors to further increase gene expression precision. Additional in-house features of the SIRION BIOTECH adeno-associated virus technology platform include surface modified particles for cell-specific transduction. Every project is thoroughly discussed with our customers to ensure that the resulting particles are feasably designed and fit the final application.
Custom AAV
The success of AAV in vivo experiments is dependent on virus purity and titer. Equally important is the correct choice of serotype and promoter.
When choosing our custom AAV service, SIRION Biotech’s technical experts will assist you to identify the optimal setting for your specific application and engineer AAV vectors exactly to your specification.
For a first step in your AAV strategy SIRION offers AAV-CMV-GFP test batches for serotype 1-6 & 1/2 for pilot experiments.
Fields of Application
- Tissue specific long term gene overexpression and knockdown in vivo
SIRION benefits
- Personal expert consultation at all stages of the project
- Strong tissue targeting with a unique serotype and promoter portfolio
- AAV delivery in less than 2 months
- Highest quality standards and prutification options to fit your experimental needs
Service Details
- Cloning of your cDNA/ shRNA into an expression vector with free choice of promoter
- Verification of cloning success by DNA-sequencing
- Small-scale test production with GO/ NO-GO decision
- GO: standard production
- Transfection of 293T cells with custom the expression vector and a serotype specific helper plasmids
- One or two-step purification and concentration inlcuding HPLC options
- Genomic titer determination, QC and Delivery
Contact us to draft your custom AAV project
AAV Configurator
Directly design your custom constructs at www.doulix.com and order SIRON AAV packaging service for full AAV particle automization.
AAV test batches
AAV for pilot experiments
The success of any AAV in vivo experiments is highly dependent on the best suitable serotype and promoter choice.
AAV serotypes are created by modifications of the capsid’s binding motif that lead to different and specific tissue tropisms. Controlled alterations can also be exploited to reduce the already low immunoegnicity of the AAV particle further. This makes AAVs a promising tool for gene therapy.
SIRION offers
- Full scientific project consulting and planning assistance for all virus related projects.
- ssAAV-CMV-GFP test batches with 1-2 X10E11GC delivered in 1-4 weeks for serotypes 1-6 & 1/2
- A broad functionality tested tissue specific promoter portfolio to enhance and optimize specificity.
* Promoters can be used for custom AAV generation and are also available as rAAV expression plasmids.
Find your best suitable serotype
The following table serves as a guideline for first AAV experiments and includes serotypes 1-6 which can be commercialized by SIRION. Recommendations are based on experimental data after systemic injection in mice as described in Zincarelli et al. 2008/ Molecular Therapy.
