Recombinant Adeno-Associated Virus Products

Recombinant adeno-associated viruses (rAAVs) are becoming the vector of choice for delivering gene therapies and have been investigated for the treatment of cancer and ocular, pancreatic and CNS diseases. Most gene therapy vectors work by integrating their genetic material into a patient’s cells; however, rAAV-delivered genes remain in the extrachromosomal state and are not incorporated into the host genome. This is an advantage, as rAAV vectors can infect both quiescent and dividing cells and are not known to cause diseases in humans.

Characterizing with Analytical Ultracentrifugation Assays

protein subunitsThis poster demonstrates analytical ultracentrifugation as a reproducible method to evaluate polydispersity in adeno-associated virus (AAV) samples and quantify AAV empty to full capsid ratios. Download Now »

Due to the complexity of rAAV therapies, Charles River has developed specialized testing packages for the characterization and quality control testing of AAV particles and constituent capsid protein assemblies. The packages include:

  • Mass Spectrometry
    • Peptide mapping
    • Intact molecular weight
    • Glycan profiling of viral envelope proteins
  • Biophysical Characterization (Higher Order Structure Analysis)
    • Analytical ultracentrifugation for determine of empty vs. full capsid ratios
    • Stability indicating methods such as DSC and DLS
  • Analytical Testing
    • RP-HPLC
    • Anion exchange HPLC
    • ELISA methods
    • Amino acid analysis
    • N-terminal sequencing

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