Heterogeneity in an adeno-associated virus transfection-based production process limits the production efficiency.

ABSTRACT: The popularity of rAAV vectors in gene therapy are placing a burden on current production systems. To improve the accessibility of these life changing treatments, increases in production yields and a reduction in the cost-of-goods are needed. Transient transfection is the most common way to introduce rAAV-encoding plasmids to producer cells but it suffers from significant drawbacks such as low and inconsistent yields as well as high cost due to its need for plasmid DNA. This study aims to address the low yield of transient transfection-based rAAV production through advanced methods in process characterization. Adherent and suspension cultures of a HEK293T cell line were triple-transfected for rAAV9 production using polyethylenimine (PEI). Samples were taken at various times post-transfecti

SUBMITTER: Ladd B 

PROVIDER: S-EPMC12586574 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature