Reversed Phase-Liquid Chromatography for Recombinant AAV Genome Integrity Assessment.

ABSTRACT: After decades of research, gene therapy products have reached market maturity in recent years. Recombinant adeno-associated viruses (rAAVs) are one of the most promising gene delivery vehicles and are currently under intense scientific investigation. These next-generation medicines remain very challenging when it comes to designing appropriate analytical techniques for quality control. One critical quality attribute is the integrity of ssDNA incorporated in these vectors. The genome is the active compound driving rAAV therapy and therefore requires proper assessment and quality control. Current techniques for rAAV genome characterization include next-generation sequencing, quantitative polymerase chain reaction, analytical ultracentrifugation (AUC), and capillary gel electrophoresis (CGE),

SUBMITTER: Gstottner C 

PROVIDER: S-EPMC10248999 | biostudies-literature | 2023 Jun

REPOSITORIES: biostudies-literature