Title:Recent Advances in the Analysis Full/Empty Capsid Ratio and Genome Integrity of Adeno-associated Virus (AAV) Gene Delivery Vectors
VOLUME: 20 ISSUE: 10
Author(s):Laszlo Hajba and Andras Guttman*
Affiliation:Translational Glycomics Research Group, Research Institute of Biomolecular and Chemical Engineering, University of Pannonia, Veszprem, Egyetem u. 10, Translational Glycomics Research Group, Research Institute of Biomolecular and Chemical Engineering, University of Pannonia, Veszprem, Egyetem u. 10
Keywords:adeno-associated virus (AAV), good manufacturing practice, full/empty capsid ratio, electron
microscopy, anion exchange chromatography, AAV vector genome integrity, high-throughput sequencing.
Abstract:Adeno-associated virus (AAV) is one of the most promising viral gene delivery
vectors with long-term gene expression and disease correction, featuring high efficiency
and excellent safety in human clinical trials. During the production of AAV vectors, there
are several quality control (QC) parameters that should be rigorously monitored to
comply with clinical safety and efficacy. This review gives a short summary of the most
frequently used AVV production and purification methods, focusing on the analytical
techniques applied to determine the full/empty capsid ratio and the integrity of the
encapsidated therapeutic DNA of the products.
