Recent Advances in the Analysis Full/Empty Capsid Ratio and Genome Integrity of Adeno-associated Virus (AAV) Gene Delivery Vectors

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,Hungary

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.

Cite this article as:

Hajba Laszlo and Guttman Andras*, Recent Advances in the Analysis Full/Empty Capsid Ratio and Genome Integrity of Adeno-associated Virus (AAV) Gene Delivery Vectors, Current Molecular Medicine 2020; 20 (10) . https://dx.doi.org/10.2174/1566524020999200730181042