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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.