Adeno-associated virus (AAV)

SIRION Biotech provides engineered adeno-associated virus (AAV) vectors for in-vivo gene expression and knockdown experiments, which help uncover the phenotypic impact of a genetic modification on the level of the living organism. AAVs are ideal for this kind of application.

Benefits of AAV include:

  • Lack of apparent pathogenicity and low immunogenicity
  • Long-term gene expression in vivo (more than one 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 fast protein liquid chromatography (FPLC) grade AAV production and engineering services, providing high-quality products in less than eight weeks. The sophisticated platform allows the integration of virtually any desired expression constructs 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.

AAV optimisation for gene therapy

SIRION BIOTECH offers a range of several different AAV serotypes in combination with tissue-specific promoters to further increase gene expression precision.

Additional in-house features of the SIRION Biotech AAV technology platform include surface modified particles for cell-specific transduction. Every project is thoroughly discussed with customers to ensure that the resulting particles are feasibly designed and fit the final application.

Custom AAV

The success of AAV in-vivo experiments is dependent on virus purity and titre. Equally important is the correct choice of serotype and promoter.

When choosing its 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 the first step in your AAV strategy, SIRION Biotech offers an AAV cytomegalovirus (CMV) green fluorescent protein (GFP) test batches for serotype 1-6 & 1/2 for pilot experiments.

SIRION Biotech’s benefits include:

  • Personal expert consultation at all stages of the project
  • Strong tissue targeting with a unique serotype and promoter portfolio
  • AAV delivery in less than two months
  • Highest quality standards and purification options to fit your experimental needs

Service details include:

  • Cloning of your complementary deoxyribonucleic acid (cDNA) / short hairpin ribonucleic acid (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
  • Transfection of 293T-cells with custom expression vector and a serotype specific helper plasmids
  • One or two-step purification and concentration, inlcuding high-performance liquid chromatography (HPLC) options
  • Genomic titre determination, quality control (QC) and delivery

AAV Configurator

SIRION Biotech offer the AAV Configurator, an online engineering and production service with the Doulix AAV Wizard.

Users directly design their custom constructs and order SIRON AAV packaging service for full AAV particle automisation.

AAV test batches

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
  • Self-complementary adeno-associated virus (ssAAV) CMV GFP test batches with 1-2 X10E11GC delivered in between one and four weeks for serotypes 1-6 and 1/2
  • A broad functionality tested tissue specific promoter portfolio to enhance and optimise specificity.

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