Vector characterization methods for quality control testing of recombinant adeno-associated viruses

Abstract

Adeno-associated virus (AAV)-based vectors expressing therapeutic gene products have shown great promise for human gene therapy. A major challenge for translation of promising research to clinical development is the establishment of appropriate quality control (QC) test methods to characterize clinical grade AAV vectors. This chapter focuses on QC testing, providing an overview of characterization methods appropriate for clinical vectors prepared for early phase clinical studies, and detailed descriptions for selected assays that are useful to assess AAV vector safety, potency, and purity.