rAAV Vector Product Characterization and Stability Studies

  • Richard O. SnyderEmail author
  • Muriel Audit
  • Joyce D. Francis
Part of the Methods in Molecular Biology book series (MIMB, volume 807)


Recombinant adeno-associated viral (rAAV) vectors mediate the safe and long-term correction of genetic diseases following a single administration. Preclinical studies in animal models and human trials have shown rAAV vector persistence and safety. In some trials, sustained or transient transgene expression has been demonstrated in humans treated for alpha-1 antitrypsin deficiency, LPL deficiency, hemophilia B and cystic fibrosis, and sustained correction of inherited blindness has been reported by three groups. For human use, rAAV vectors are manufactured and tested in compliance with current Good Manufacturing Practices as outlined in the Code of Federal Regulations (21CFR) or European Good Manufacturing Practices (Eudralex, Volume 4, GMP Guidelines, 2003/94/CE and 91/356/EEC). Manufacturing control, as well as product quality is evaluated by quality control testing and all manufacturing, facilities, and testing activities are reviewed by the quality assurance department. In-process specifications are set and in-process testing is conducted to confirm that the manufacturing process is controlled, aseptic, and performs consistently. Final product is tested to ensure release specifications are met for identity, safety, purity, potency, and stability.

Key words

Adeno-associated virus Gene therapy Assay Testing Quality control Quality assurance 



We acknowledge Mahuya Bose and Weiyi Ni for insightful discussions. ROS is an inventor on patents related to recombinant AAV technology and owns equity in a gene therapy company that is commercializing AAV for gene therapy applications. To the extent that the work in this manuscript increases the value of these commercial holdings ROS has a conflict of interest.


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Copyright information

© Springer Science+Business Media, LLC 2012

Authors and Affiliations

  • Richard O. Snyder
    • 1
    • 3
    Email author
  • Muriel Audit
    • 2
  • Joyce D. Francis
    • 3
  1. 1.Department of Molecular Genetics and MicrobiologyUniversity of Florida College of MedicineGainesvilleUSA
  2. 2.GenoSafe SASEVRY CedexFrance
  3. 3.Center of Excellence for Regenerative Health BiotechnologyUniversity of FloridaAlachuaUSA

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