Methodological validation of a cryo-TEM-based detection method for empty capsid ratio in recombinant adeno-associated virus.

ABSTRACT: Recombinant adeno-associated virus (rAAV) is one of the most promising vectors for gene therapy. It consists of a protein capsid that encapsulates the genetic material. However, during the production process, except for the full particles containing the genome, a variety of types of particles are also produced concomitantly, such as empty particles (no DNA encapsulated in the capsid), viral fragments, damaged viruses, and viral aggregates. Empty particles have been reported to induce unnecessary immune response and reduce transduction efficiency. Therefore, a quantitative method that objectively assesses the content of rAAV particles containing the genome is crucial for quality measurement. In this study, we developed a technique to detect the proportion of empty capsids in rAAV samples by

SUBMITTER: Xu L 

PROVIDER: S-EPMC12750778 | biostudies-literature | 2026 Mar

REPOSITORIES: biostudies-literature